Publications
Explore some of our selected publications
What is the consistency of response to rimegepant at a group level in the acute treatment of migraine in UK adult patients? Protocol for a real-world, patient-centred study
O’Neil G, Abraham L, Pawinski R, Nakajima K, Bagshaw E, Fellows A, Llewellyn S, Lambru G (EHC, 2025)
Assessment of stable chronic bronchitis improvement with adjunctive long-term Mucinex® use via the cough and sputum assessment questionnaire (CASA-Q)
Divel C, Borecka O, Llewellyn S, Spangenthal S (CHEST, 2025)
Adjunctive long-term use of Mucinex® leading to improvement in stable chronic bronchitis and patient’s quality of life: A case report.
Spangenthal S, Divel C, Borecka O, Llewellyn S (Medical Reports, 2025)
Real-world use of Vitaccess Real™ platform to assess quality of life impact with long-term use of Mucinex® in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Llewellyn S (ISOQOL, 2025)
Multiple myeloma caregiver costs and disabilities data for economic modelling and HTA submissions
Alsawady M, Tarnowska R, Kudlac A, Vincent S, Melrose D, Lied-Lied A (ISPOR Europe, 2025)
A targeted review of caregiver experiences of CAR-T therapy in ambulatory settings
Ringland C, Bagshaw E, Llewellyn S, Pugh G (ISPOR Europe, 2025)
Extrapulmonary disease burden and impact of cystic fibrosis (CF) on productivity in people with CF (pwCF) aged >12 years not treated with CF transmembrane conductance regulator modulators (CFTRm): interim analysis (IA) of the HUBBLE study
Elborn S, Mainz J, Abbott J, Carr S, Sole A, Costa S, Ganapathy V, Arteaga-Solis E, Liu J, Yuan J, Thorat T, Llewellyn S, Larkin M, De Iorio F, Kaplowitz H (ITS Annual Scientific Meeting, 2021)
Patient‑ and Caregiver‑Reported Burden of Transfusion‑Dependent β‑Thalassemia Measured Using a Digital Application
Paramore C, Levine L, Bagshaw E, Ouyang C, Kudlac A, Larkin M (Springer Nature’s, The Patient – Patient-Centered Outcomes Research, 2020)
Patient-reported burden of transfusion-dependent Beta-Thalassemia in the USA and UK measured using a digital app
Paramore C, Helfer J, Levine L, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Deprivation and access to care among patients with melanoma in the UK
Quinn C, Larkin M, Jacobs I, Kandola S, Smoyer K (ISPOR EU, 2022)
Using patient-reported data to estimate costs associated with melanoma
Javed M, Quinn C, Amini F, Boxell E (National PROMs Annual Research Conference, 2022)
Symptoms and side effects associated with melanoma treatment
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL 2021)
Contextualizing qualitative analysis with quantitative data on the symptoms and side effects associated with melanoma treatment in the real world – the value of patient registries
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL, 2021)
How do patients manage side effects during melanoma treatment? The importance of social support and doctor–patient communication
Ofori A, Hall K, Bottomley C, Larkin M, Heinrich M (The Christie School of Oncology, Melanoma Study Day, 2021)
Rewarding participants through charitable donations to Melanoma UK motivates recruitment and engagement: a bring your own device study example
Hall K, Ouyang C, Chilongo A, Llewellyn S, Quinn C, Larkin M (Quality of Life Research; 29: S91-S92)
Patient-centricity in action: using patient feedback to adapt the PRO-CTCAE instrument in a real-world study
Llewellyn S, Hall K, Camidge L, Bottomley C, Nixon A, Larkin M (Quality of Life Research, 29: S91)
Melanoma patient registries: a targeted review and comparison of datasets
Hall K, Llewellyn S, Bottomley C, Larkin M (Quality of Life Research, 29: S113)
Assessing the value of real-world evidence in melanoma in health technology assessment appraisals
Llewellyn S, Doe A, Quinn C (DIA Europe, 2020)
Targeted review of adverse events associated with treatments for stage 3 and 4 melanoma
Llewellyn S, Bagshaw E, Åkesson C, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Adverse events associated with immunotherapies used in the treatment of stage 3 and 4 melanoma
Llewellyn S, Åkesson C, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for melanoma in the UK measured using a digital ‘bring your own device’ platform
Larkin J, Cannon D, Nuttall G, Au L, Hunter N, Spain L, Turajlic S, Åkesson C, Llewellyn S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2019)
Real-world HRQL in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (PROMS Conference, 2018)
Taking the burden out of symptom reporting in patients with melanoma using a digital real-world evidence platform
Au L, Nuttall G, Cannon D, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Larkin J, Wiseman T (ISPOR US, 2018)
Real-world EQ-5D-5L utility values in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2018)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-reported severity of pain interference in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (ANNEM 2023)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Lower extremity + upper limb disability in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A
Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray AJ, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Sénéchal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 2022 24(1): 7–17 )
Depression in patients with Charcot-Marie-Tooth
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Patient-reported symptom burden of Charcot-Marie-Tooth_PNS_2022
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Work impacts in patients with Charcot-Marie-Tooth_PNS_2022
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Disease burden and quality of life in patients with Charcot-Marie-Tooth disease type 1A, 2, 4 and X in Europe and the US measured using a digital app
Ziemssen T, Thomas FP, Sivera R, Saporta M, Genovese F, Gray A, Hollett C, Moore A, Rego M, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Larkin M, Ouyang C, Quinn C (Muscle & Nerve, 62: S69-S70)
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study
Thomas FP, Saporta M, Attarian S, Sevilla T, Mascaró RS, Fabrizi GM, Genovese F, Gray A, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Monteiro K, Paoli X, Llewellyn S, Larkin M, Boutalbi Y (Neurodegenerative Disease Management, 2021; 11(1): 21-33)
Patient-reported symptom burden of Charcot-Marie-Tooth disease Type 1a in Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343)
Patient-reported burden of Charcot-Marie-Tooth disease Type 1a In Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343-344)
Productivity losses in people with Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world evidence study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Healthcare resource use in Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Demographics and treatment patterns for Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Diversity in the Charcot–Marie–Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment of the Charcot-Marie-Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Monteiro K, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment patterns for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Care standards for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Diversity in the Charcot-Marie-Tooth disease population in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Digitally assessed real-world diversity in people with Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Digitally assessed patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Development of a digital app and innovative recruitment for an international real-world observational study in Charcot-Marie-Tooth disease
Ziemssen T, Attarian S, Thomas FP, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Kousoulakou H, Larkin M (ISPOR EU, 2018)
Patient-reported impact of adult-onset ALSP
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Caregiving burden among caregivers of people with myasthenia gravis
Dewilde S, Tollenaar NH, Boulanger P, Archer A, Pardo R, Cortés-Vicente E, Mantegazza R, Vanoli F, Lehnerer S, Pawlitzki M, Heinrich M, De Ruyck F, Phillips G, Paci S (Orphanet Journal of Rare Diseases, 2025)
Harnessing digital measures of physical function: findings from a targeted literature review
Aggio D, Ringland C, Amini F, Bowmar E, Llewellyn S (ISPOR, 2025)
A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry
Amini F, Lawrence J, Vincent S, Penduka T, Hall G, Fellows A, Larkin M, Scowcroft A, Beau Lejdstrom R, Savic N, Jacob S, Habib A, Saccà F, Hayes A (AAN, 2025 and MGFA International, 2025)
The interplay between CASA-Q domains: insights from a real-world study investigating the effects of extended-release guaifenesin in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Fellows A, Llewellyn S (ACC, 2025)
Adjunctive long-term Mucinex® use leading to improvement in stable chronic bronchitis and decreased health care resource utilization – a case report
Divel C, Borecka O, Llewellyn S, Patel P, Shea T, Adeleke M, Spangenthal S (ATS, 2025)
Modified Delphi panel on advanced ALK-positive NSCLC patient profiling, treatment landscape, and clinical guidelines in the UK
Patel A, Tsang C, Baig LE, Llewellyn S, Baijal S (BTOG, 2025)
Caregiver-reported quality of life of patients with MCT8 deficiency: Results from a cross-sectional survey
Ofori A, Larkin M, Georges N (ISPOR Europe, 2024)
Quality of Life Impact of Caregiving for Patients with MCT8 Deficiency: Results from a Cross-Sectional Survey
Ofori A, Larkin M, Georges N (ISPOR EU, 2024)
Quality of patient engagement activities in health economics and outcomes research: insights from the ISPOR community
Llewellyn S, Qiao N, Masurkar P, Botto-van Bemden A, Roydhouse J, Perfetto E (Value & Outcomes Spotlight, 10(6):41-45)
Development of two conceptual models to describe the patient and caregiver reported impacts of living with X-linked myotubular myopathy (XLMTM)
Borecka O, Lawrence J, Llewellyn S (ISOQOL, 2024)
Leveraging SMS for patient-reported data collection
Llewellyn S, Amini F (ISOQOL, 2024)
Duchenne muscular dystrophy (DMD) patient vignettes development methodology
Merla V, Posner N, Borecka O, Vincent S (EAN, 2024)
Real world effectiveness of guaifenesin ER in tackling mucus hypersecretion in stable chronic bronchitis
Spangenthal S, Llewellyn S, Borecka O, Pollack C, Adeleke M, Kulasekaran A, Birring S, Mazzone S, Shea T (The Thirteenth London International Cough Symposium, 2024)
Patient-reported outcome measures used in interventional studies of irritable bowel syndrome: a targeted review
Lawrence J, Boxell E (National PROMS UK, 2024)
Outcomes and interventions in clinical studies investigating pharmacological therapies for the treatment of transthyretin amyloidosis: a targeted review
Alsawady M, Borecka O (National PROMS UK, 2024)
Diagnosis Validation in Patient-Centered Research
Alsawady M, Lawrence J, Amini F, Vincent S (Value and Outcomes Spotlight, 2024)
Assessing adherence in real-world studies
Ofori A, Octavia Borecka O, Llewellyn S
Approaches and challenges in developing caregiver vignettes in rare, progressive, and fatal childhood conditions: learnings from vignette development in Duchenne muscular dystrophy (DMD)
Llewellyn S, Borecka O, Bottomley C, Richardson L, Biggane A (ISOQOL, 2023)
Caregiver burden in growth hormone deficiency: a targeted review
Amini F, Alsawady M, Borecka O, Bottomley C
(ISPOR EU, 2023)
“I’ve got a text!” Feedback from prospective participants on the use of SMS surveys in real-world studies
Lawrence J, Amini F, Monera-Penduka TG, Llewellyn S
(ISPOR EU, 2023)
Vitamin D as an adjunct therapy in the treatment of atopic dermatitis: a targeted review
Borecka O, Lawrence J, Llewellyn S (ISPOR EU, 2023)
A targeted review of pharmacological therapies for the treatment of chronic spontaneous urticaria
Lawrence J, Borecka O, Alsawady M, Sauca M, Kallmes K, Llewellyn S (ISPOR EU, 2023)
Adverse events associated with clinical studies
Alsawady M, Borecka O & Llewellyn S (ISPOR US, 2023)
Patient feedback on the use of reward programs
Alsawady M, Amini F, Vincent S, Llewellyn S (ISPOR US, 2023)
Effectiveness of prostate cancer screening
Llewellyn S (ISPOR US, 2023)
Engagement statistics in real-world studies
Day L, Lau J, Quinn C (ISPOR EU, 2022)
Adherence rates in real-world studies
Borecka O, Ofori A & Llewellyn S (ISPOR EU, 2022)
Digital diagnosis validation feature
Vincent S, Borecka O, Llewellyn S & Bottomley C (ISPOR EU, 2022)
White paper: Patient-centric real-world evidence
The power of technology in real-world evidence generation
In today’s healthcare market, patient-centric evidence is crucial for product differentiation and success.
Diagnosis validation in patient-centered research
Llewellyn S, Bottomley C, Borecka O (National PROMs Annual Research Conference, 2022)
Patient-centricity with digital real-world evidence
When it comes to drug discovery there is a wealth of insight to be gained from the patient community. Involving patients in the design and development of real-world evidence studies, which in turn complement traditional clinical trials by demonstrating patients’ lived experience of the condition and its treatments, can be a powerful means of emphasizing patient-centricity in drug development. In this article, the authors explore the technicalities of engaging patients in real-world evidence studies.
Building a bridge between patient and pharma
To what extent does the scientific relevance of a healthcare research question parallel its relevance from the patient’s perspective? In this article, we use a live example of a real-world study investigating the experiences of individuals living with Charcot-Marie-Tooth disease (CMT) to demonstrate the wide-reaching value of patient involvement in study design and development.
Reimbursement of digital therapeutics
A comparison of the reimbursement of digital therapeutics in France, Germany, and the UK. Presented at ISPOR EU 2021.
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (Global Genes RARE Patient Advocacy Summit, 2022)
Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)
This manuscript was published in BMJ Open in May 2023. It discusses the patient-reported impact of myasthenia gravis in the real world using findings from a digital observational survey-based study (MyRealWorld MG).See here for more of our publications.Author Author...
Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG
Dewilde S, Philips G, Paci S, Beauchamp J, Chiroli S, Quinn C, Day L, Larkin M, Palace J, Berrih-Aknin S, Claeys K, Muppidi S, Mantegazza R, Saccà F, Meisel A, Bassez G, Murai H, Janssen MF (BMJ Open, 2022)
WHITE PAPER: Unleashing the potential of real-world evidence in rare diseases
Murthy A, Mantegazza R, Larkin M, Friconneau M, Annemans L, Van Damme B, Sokol T, Emond S (2022)
Digital data collection to measure the impact of myasthenia gravis on patients’ utility values in the real world
Dewilde S, Kousoulakou H, Janssen M, Claeys K, Friconneau M, Jacob S, Meisel A, Day L, Quinn C, Larkin M, Leighton T, Phillips G, Paci S (Value in Health, 25(1): S246)
Real-world patient-reported impact of myasthenia gravis: initial data from the MyRealWorld™ MG study
This poster was presented in October 2021 at the AANEM annual meeting. It shares the initial data from the MyRealWorld™ MG study about the impact of myasthenia gravis on the daily lives of patients.See here for more of our publications on myasthenia gravis and other...
Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
Berrih-Aknin S, Claeys K, Law N, Mantegazza R, Murai H, Sacca F, Dewilde S, Janssen M, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (BMJ Open 2021;11:e048198. doi: 10.1136/bmjopen-2020-048198)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Assouly P, Berrih-Aknin S, Claeys K, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J (Muscle & Nerve, 62: S65-S66)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Palace J, Assouly P, Claeys KG, Saccà F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (ABN, 2020)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Meisel A, Assouly P, Berrih-Aknin S, Claeys KG, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (DGN, 2020)
What role does caregiver burden play in health technology assessment bodies’ appraisals of highly specialized technologies?
Ofori A, Llewellyn S, Bottomley C, Kousoulakou H (ISPOR EU, 2020)
Sickle cell disease patient registries are not collecting enough data
Llewellyn S, Doe A, Åkesson C, Kousoulakou H, Quinn C, Larkin M (ISPOR US, 2020)
Complexities in localizing participant profile survey questions in real-world studies for smartphone apps
Kretz S, Richards A (Quality of Life Research, 29: S135)
The clinical and cost-effectiveness of supplemental parenteral nutrition in oncology
Webb N, Fricke J, Hancock E, Trueman D, Ghosh S, Winstone J, Miners A, Shepelev J, Valle JW (ESMO Open. 2020 Jun 1;5(3):e000709)
Challenges with using paper format legacy Patient Reported Outcome Measures (PROMs) in digital real-world evidence (RWE) studies for apps
Kretz S, Spinage C, Bottomley C (Quality of Life Research, 29: S137)
Innovative partnerships with patients and patient advocacy groups in digital observational rare disease studies.
Llewellyn S,Bagshaw E,Williams H,Larkin M (RARE Patient Advocacy Summit; 2019 Sep 18-20; San Diego, CA, US)
Myasthenia gravis
What is the consistency of response to rimegepant at a group level in the acute treatment of migraine in UK adult patients? Protocol for a real-world, patient-centred study
O’Neil G, Abraham L, Pawinski R, Nakajima K, Bagshaw E, Fellows A, Llewellyn S, Lambru G (EHC, 2025)
Assessment of stable chronic bronchitis improvement with adjunctive long-term Mucinex® use via the cough and sputum assessment questionnaire (CASA-Q)
Divel C, Borecka O, Llewellyn S, Spangenthal S (CHEST, 2025)
Adjunctive long-term use of Mucinex® leading to improvement in stable chronic bronchitis and patient’s quality of life: A case report.
Spangenthal S, Divel C, Borecka O, Llewellyn S (Medical Reports, 2025)
Real-world use of Vitaccess Real™ platform to assess quality of life impact with long-term use of Mucinex® in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Llewellyn S (ISOQOL, 2025)
Multiple myeloma caregiver costs and disabilities data for economic modelling and HTA submissions
Alsawady M, Tarnowska R, Kudlac A, Vincent S, Melrose D, Lied-Lied A (ISPOR Europe, 2025)
A targeted review of caregiver experiences of CAR-T therapy in ambulatory settings
Ringland C, Bagshaw E, Llewellyn S, Pugh G (ISPOR Europe, 2025)
Extrapulmonary disease burden and impact of cystic fibrosis (CF) on productivity in people with CF (pwCF) aged >12 years not treated with CF transmembrane conductance regulator modulators (CFTRm): interim analysis (IA) of the HUBBLE study
Elborn S, Mainz J, Abbott J, Carr S, Sole A, Costa S, Ganapathy V, Arteaga-Solis E, Liu J, Yuan J, Thorat T, Llewellyn S, Larkin M, De Iorio F, Kaplowitz H (ITS Annual Scientific Meeting, 2021)
Patient‑ and Caregiver‑Reported Burden of Transfusion‑Dependent β‑Thalassemia Measured Using a Digital Application
Paramore C, Levine L, Bagshaw E, Ouyang C, Kudlac A, Larkin M (Springer Nature’s, The Patient – Patient-Centered Outcomes Research, 2020)
Patient-reported burden of transfusion-dependent Beta-Thalassemia in the USA and UK measured using a digital app
Paramore C, Helfer J, Levine L, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Deprivation and access to care among patients with melanoma in the UK
Quinn C, Larkin M, Jacobs I, Kandola S, Smoyer K (ISPOR EU, 2022)
Using patient-reported data to estimate costs associated with melanoma
Javed M, Quinn C, Amini F, Boxell E (National PROMs Annual Research Conference, 2022)
Symptoms and side effects associated with melanoma treatment
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL 2021)
Contextualizing qualitative analysis with quantitative data on the symptoms and side effects associated with melanoma treatment in the real world – the value of patient registries
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL, 2021)
How do patients manage side effects during melanoma treatment? The importance of social support and doctor–patient communication
Ofori A, Hall K, Bottomley C, Larkin M, Heinrich M (The Christie School of Oncology, Melanoma Study Day, 2021)
Rewarding participants through charitable donations to Melanoma UK motivates recruitment and engagement: a bring your own device study example
Hall K, Ouyang C, Chilongo A, Llewellyn S, Quinn C, Larkin M (Quality of Life Research; 29: S91-S92)
Patient-centricity in action: using patient feedback to adapt the PRO-CTCAE instrument in a real-world study
Llewellyn S, Hall K, Camidge L, Bottomley C, Nixon A, Larkin M (Quality of Life Research, 29: S91)
Melanoma patient registries: a targeted review and comparison of datasets
Hall K, Llewellyn S, Bottomley C, Larkin M (Quality of Life Research, 29: S113)
Assessing the value of real-world evidence in melanoma in health technology assessment appraisals
Llewellyn S, Doe A, Quinn C (DIA Europe, 2020)
Targeted review of adverse events associated with treatments for stage 3 and 4 melanoma
Llewellyn S, Bagshaw E, Åkesson C, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Adverse events associated with immunotherapies used in the treatment of stage 3 and 4 melanoma
Llewellyn S, Åkesson C, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for melanoma in the UK measured using a digital ‘bring your own device’ platform
Larkin J, Cannon D, Nuttall G, Au L, Hunter N, Spain L, Turajlic S, Åkesson C, Llewellyn S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2019)
Real-world HRQL in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (PROMS Conference, 2018)
Taking the burden out of symptom reporting in patients with melanoma using a digital real-world evidence platform
Au L, Nuttall G, Cannon D, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Larkin J, Wiseman T (ISPOR US, 2018)
Real-world EQ-5D-5L utility values in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2018)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-reported severity of pain interference in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (ANNEM 2023)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Lower extremity + upper limb disability in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A
Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray AJ, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Sénéchal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 2022 24(1): 7–17 )
Depression in patients with Charcot-Marie-Tooth
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Patient-reported symptom burden of Charcot-Marie-Tooth_PNS_2022
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Work impacts in patients with Charcot-Marie-Tooth_PNS_2022
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Disease burden and quality of life in patients with Charcot-Marie-Tooth disease type 1A, 2, 4 and X in Europe and the US measured using a digital app
Ziemssen T, Thomas FP, Sivera R, Saporta M, Genovese F, Gray A, Hollett C, Moore A, Rego M, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Larkin M, Ouyang C, Quinn C (Muscle & Nerve, 62: S69-S70)
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study
Thomas FP, Saporta M, Attarian S, Sevilla T, Mascaró RS, Fabrizi GM, Genovese F, Gray A, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Monteiro K, Paoli X, Llewellyn S, Larkin M, Boutalbi Y (Neurodegenerative Disease Management, 2021; 11(1): 21-33)
Patient-reported symptom burden of Charcot-Marie-Tooth disease Type 1a in Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343)
Patient-reported burden of Charcot-Marie-Tooth disease Type 1a In Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343-344)
Productivity losses in people with Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world evidence study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Healthcare resource use in Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Demographics and treatment patterns for Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Diversity in the Charcot–Marie–Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment of the Charcot-Marie-Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Monteiro K, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment patterns for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Care standards for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Diversity in the Charcot-Marie-Tooth disease population in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Digitally assessed real-world diversity in people with Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Digitally assessed patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Development of a digital app and innovative recruitment for an international real-world observational study in Charcot-Marie-Tooth disease
Ziemssen T, Attarian S, Thomas FP, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Kousoulakou H, Larkin M (ISPOR EU, 2018)
Patient-reported impact of adult-onset ALSP
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Caregiving burden among caregivers of people with myasthenia gravis
Dewilde S, Tollenaar NH, Boulanger P, Archer A, Pardo R, Cortés-Vicente E, Mantegazza R, Vanoli F, Lehnerer S, Pawlitzki M, Heinrich M, De Ruyck F, Phillips G, Paci S (Orphanet Journal of Rare Diseases, 2025)
Harnessing digital measures of physical function: findings from a targeted literature review
Aggio D, Ringland C, Amini F, Bowmar E, Llewellyn S (ISPOR, 2025)
A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry
Amini F, Lawrence J, Vincent S, Penduka T, Hall G, Fellows A, Larkin M, Scowcroft A, Beau Lejdstrom R, Savic N, Jacob S, Habib A, Saccà F, Hayes A (AAN, 2025 and MGFA International, 2025)
The interplay between CASA-Q domains: insights from a real-world study investigating the effects of extended-release guaifenesin in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Fellows A, Llewellyn S (ACC, 2025)
Adjunctive long-term Mucinex® use leading to improvement in stable chronic bronchitis and decreased health care resource utilization – a case report
Divel C, Borecka O, Llewellyn S, Patel P, Shea T, Adeleke M, Spangenthal S (ATS, 2025)
Modified Delphi panel on advanced ALK-positive NSCLC patient profiling, treatment landscape, and clinical guidelines in the UK
Patel A, Tsang C, Baig LE, Llewellyn S, Baijal S (BTOG, 2025)
Caregiver-reported quality of life of patients with MCT8 deficiency: Results from a cross-sectional survey
Ofori A, Larkin M, Georges N (ISPOR Europe, 2024)
Quality of Life Impact of Caregiving for Patients with MCT8 Deficiency: Results from a Cross-Sectional Survey
Ofori A, Larkin M, Georges N (ISPOR EU, 2024)
Quality of patient engagement activities in health economics and outcomes research: insights from the ISPOR community
Llewellyn S, Qiao N, Masurkar P, Botto-van Bemden A, Roydhouse J, Perfetto E (Value & Outcomes Spotlight, 10(6):41-45)
Development of two conceptual models to describe the patient and caregiver reported impacts of living with X-linked myotubular myopathy (XLMTM)
Borecka O, Lawrence J, Llewellyn S (ISOQOL, 2024)
Leveraging SMS for patient-reported data collection
Llewellyn S, Amini F (ISOQOL, 2024)
Duchenne muscular dystrophy (DMD) patient vignettes development methodology
Merla V, Posner N, Borecka O, Vincent S (EAN, 2024)
Real world effectiveness of guaifenesin ER in tackling mucus hypersecretion in stable chronic bronchitis
Spangenthal S, Llewellyn S, Borecka O, Pollack C, Adeleke M, Kulasekaran A, Birring S, Mazzone S, Shea T (The Thirteenth London International Cough Symposium, 2024)
Patient-reported outcome measures used in interventional studies of irritable bowel syndrome: a targeted review
Lawrence J, Boxell E (National PROMS UK, 2024)
Outcomes and interventions in clinical studies investigating pharmacological therapies for the treatment of transthyretin amyloidosis: a targeted review
Alsawady M, Borecka O (National PROMS UK, 2024)
Diagnosis Validation in Patient-Centered Research
Alsawady M, Lawrence J, Amini F, Vincent S (Value and Outcomes Spotlight, 2024)
Assessing adherence in real-world studies
Ofori A, Octavia Borecka O, Llewellyn S
Approaches and challenges in developing caregiver vignettes in rare, progressive, and fatal childhood conditions: learnings from vignette development in Duchenne muscular dystrophy (DMD)
Llewellyn S, Borecka O, Bottomley C, Richardson L, Biggane A (ISOQOL, 2023)
Caregiver burden in growth hormone deficiency: a targeted review
Amini F, Alsawady M, Borecka O, Bottomley C
(ISPOR EU, 2023)
“I’ve got a text!” Feedback from prospective participants on the use of SMS surveys in real-world studies
Lawrence J, Amini F, Monera-Penduka TG, Llewellyn S
(ISPOR EU, 2023)
Vitamin D as an adjunct therapy in the treatment of atopic dermatitis: a targeted review
Borecka O, Lawrence J, Llewellyn S (ISPOR EU, 2023)
A targeted review of pharmacological therapies for the treatment of chronic spontaneous urticaria
Lawrence J, Borecka O, Alsawady M, Sauca M, Kallmes K, Llewellyn S (ISPOR EU, 2023)
Adverse events associated with clinical studies
Alsawady M, Borecka O & Llewellyn S (ISPOR US, 2023)
Patient feedback on the use of reward programs
Alsawady M, Amini F, Vincent S, Llewellyn S (ISPOR US, 2023)
Effectiveness of prostate cancer screening
Llewellyn S (ISPOR US, 2023)
Engagement statistics in real-world studies
Day L, Lau J, Quinn C (ISPOR EU, 2022)
Adherence rates in real-world studies
Borecka O, Ofori A & Llewellyn S (ISPOR EU, 2022)
Digital diagnosis validation feature
Vincent S, Borecka O, Llewellyn S & Bottomley C (ISPOR EU, 2022)
White paper: Patient-centric real-world evidence
The power of technology in real-world evidence generation
In today’s healthcare market, patient-centric evidence is crucial for product differentiation and success.
Diagnosis validation in patient-centered research
Llewellyn S, Bottomley C, Borecka O (National PROMs Annual Research Conference, 2022)
Patient-centricity with digital real-world evidence
When it comes to drug discovery there is a wealth of insight to be gained from the patient community. Involving patients in the design and development of real-world evidence studies, which in turn complement traditional clinical trials by demonstrating patients’ lived experience of the condition and its treatments, can be a powerful means of emphasizing patient-centricity in drug development. In this article, the authors explore the technicalities of engaging patients in real-world evidence studies.
Building a bridge between patient and pharma
To what extent does the scientific relevance of a healthcare research question parallel its relevance from the patient’s perspective? In this article, we use a live example of a real-world study investigating the experiences of individuals living with Charcot-Marie-Tooth disease (CMT) to demonstrate the wide-reaching value of patient involvement in study design and development.
Reimbursement of digital therapeutics
A comparison of the reimbursement of digital therapeutics in France, Germany, and the UK. Presented at ISPOR EU 2021.
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (Global Genes RARE Patient Advocacy Summit, 2022)
Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)
This manuscript was published in BMJ Open in May 2023. It discusses the patient-reported impact of myasthenia gravis in the real world using findings from a digital observational survey-based study (MyRealWorld MG).See here for more of our publications.Author Author...
Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG
Dewilde S, Philips G, Paci S, Beauchamp J, Chiroli S, Quinn C, Day L, Larkin M, Palace J, Berrih-Aknin S, Claeys K, Muppidi S, Mantegazza R, Saccà F, Meisel A, Bassez G, Murai H, Janssen MF (BMJ Open, 2022)
WHITE PAPER: Unleashing the potential of real-world evidence in rare diseases
Murthy A, Mantegazza R, Larkin M, Friconneau M, Annemans L, Van Damme B, Sokol T, Emond S (2022)
Digital data collection to measure the impact of myasthenia gravis on patients’ utility values in the real world
Dewilde S, Kousoulakou H, Janssen M, Claeys K, Friconneau M, Jacob S, Meisel A, Day L, Quinn C, Larkin M, Leighton T, Phillips G, Paci S (Value in Health, 25(1): S246)
Real-world patient-reported impact of myasthenia gravis: initial data from the MyRealWorld™ MG study
This poster was presented in October 2021 at the AANEM annual meeting. It shares the initial data from the MyRealWorld™ MG study about the impact of myasthenia gravis on the daily lives of patients.See here for more of our publications on myasthenia gravis and other...
Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
Berrih-Aknin S, Claeys K, Law N, Mantegazza R, Murai H, Sacca F, Dewilde S, Janssen M, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (BMJ Open 2021;11:e048198. doi: 10.1136/bmjopen-2020-048198)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Assouly P, Berrih-Aknin S, Claeys K, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J (Muscle & Nerve, 62: S65-S66)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Palace J, Assouly P, Claeys KG, Saccà F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (ABN, 2020)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Meisel A, Assouly P, Berrih-Aknin S, Claeys KG, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (DGN, 2020)
What role does caregiver burden play in health technology assessment bodies’ appraisals of highly specialized technologies?
Ofori A, Llewellyn S, Bottomley C, Kousoulakou H (ISPOR EU, 2020)
Sickle cell disease patient registries are not collecting enough data
Llewellyn S, Doe A, Åkesson C, Kousoulakou H, Quinn C, Larkin M (ISPOR US, 2020)
Complexities in localizing participant profile survey questions in real-world studies for smartphone apps
Kretz S, Richards A (Quality of Life Research, 29: S135)
The clinical and cost-effectiveness of supplemental parenteral nutrition in oncology
Webb N, Fricke J, Hancock E, Trueman D, Ghosh S, Winstone J, Miners A, Shepelev J, Valle JW (ESMO Open. 2020 Jun 1;5(3):e000709)
Challenges with using paper format legacy Patient Reported Outcome Measures (PROMs) in digital real-world evidence (RWE) studies for apps
Kretz S, Spinage C, Bottomley C (Quality of Life Research, 29: S137)
Innovative partnerships with patients and patient advocacy groups in digital observational rare disease studies.
Llewellyn S,Bagshaw E,Williams H,Larkin M (RARE Patient Advocacy Summit; 2019 Sep 18-20; San Diego, CA, US)
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)
What is the consistency of response to rimegepant at a group level in the acute treatment of migraine in UK adult patients? Protocol for a real-world, patient-centred study
O’Neil G, Abraham L, Pawinski R, Nakajima K, Bagshaw E, Fellows A, Llewellyn S, Lambru G (EHC, 2025)
Assessment of stable chronic bronchitis improvement with adjunctive long-term Mucinex® use via the cough and sputum assessment questionnaire (CASA-Q)
Divel C, Borecka O, Llewellyn S, Spangenthal S (CHEST, 2025)
Adjunctive long-term use of Mucinex® leading to improvement in stable chronic bronchitis and patient’s quality of life: A case report.
Spangenthal S, Divel C, Borecka O, Llewellyn S (Medical Reports, 2025)
Real-world use of Vitaccess Real™ platform to assess quality of life impact with long-term use of Mucinex® in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Llewellyn S (ISOQOL, 2025)
Multiple myeloma caregiver costs and disabilities data for economic modelling and HTA submissions
Alsawady M, Tarnowska R, Kudlac A, Vincent S, Melrose D, Lied-Lied A (ISPOR Europe, 2025)
A targeted review of caregiver experiences of CAR-T therapy in ambulatory settings
Ringland C, Bagshaw E, Llewellyn S, Pugh G (ISPOR Europe, 2025)
Extrapulmonary disease burden and impact of cystic fibrosis (CF) on productivity in people with CF (pwCF) aged >12 years not treated with CF transmembrane conductance regulator modulators (CFTRm): interim analysis (IA) of the HUBBLE study
Elborn S, Mainz J, Abbott J, Carr S, Sole A, Costa S, Ganapathy V, Arteaga-Solis E, Liu J, Yuan J, Thorat T, Llewellyn S, Larkin M, De Iorio F, Kaplowitz H (ITS Annual Scientific Meeting, 2021)
Patient‑ and Caregiver‑Reported Burden of Transfusion‑Dependent β‑Thalassemia Measured Using a Digital Application
Paramore C, Levine L, Bagshaw E, Ouyang C, Kudlac A, Larkin M (Springer Nature’s, The Patient – Patient-Centered Outcomes Research, 2020)
Patient-reported burden of transfusion-dependent Beta-Thalassemia in the USA and UK measured using a digital app
Paramore C, Helfer J, Levine L, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Deprivation and access to care among patients with melanoma in the UK
Quinn C, Larkin M, Jacobs I, Kandola S, Smoyer K (ISPOR EU, 2022)
Using patient-reported data to estimate costs associated with melanoma
Javed M, Quinn C, Amini F, Boxell E (National PROMs Annual Research Conference, 2022)
Symptoms and side effects associated with melanoma treatment
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL 2021)
Contextualizing qualitative analysis with quantitative data on the symptoms and side effects associated with melanoma treatment in the real world – the value of patient registries
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL, 2021)
How do patients manage side effects during melanoma treatment? The importance of social support and doctor–patient communication
Ofori A, Hall K, Bottomley C, Larkin M, Heinrich M (The Christie School of Oncology, Melanoma Study Day, 2021)
Rewarding participants through charitable donations to Melanoma UK motivates recruitment and engagement: a bring your own device study example
Hall K, Ouyang C, Chilongo A, Llewellyn S, Quinn C, Larkin M (Quality of Life Research; 29: S91-S92)
Patient-centricity in action: using patient feedback to adapt the PRO-CTCAE instrument in a real-world study
Llewellyn S, Hall K, Camidge L, Bottomley C, Nixon A, Larkin M (Quality of Life Research, 29: S91)
Melanoma patient registries: a targeted review and comparison of datasets
Hall K, Llewellyn S, Bottomley C, Larkin M (Quality of Life Research, 29: S113)
Assessing the value of real-world evidence in melanoma in health technology assessment appraisals
Llewellyn S, Doe A, Quinn C (DIA Europe, 2020)
Targeted review of adverse events associated with treatments for stage 3 and 4 melanoma
Llewellyn S, Bagshaw E, Åkesson C, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Adverse events associated with immunotherapies used in the treatment of stage 3 and 4 melanoma
Llewellyn S, Åkesson C, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for melanoma in the UK measured using a digital ‘bring your own device’ platform
Larkin J, Cannon D, Nuttall G, Au L, Hunter N, Spain L, Turajlic S, Åkesson C, Llewellyn S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2019)
Real-world HRQL in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (PROMS Conference, 2018)
Taking the burden out of symptom reporting in patients with melanoma using a digital real-world evidence platform
Au L, Nuttall G, Cannon D, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Larkin J, Wiseman T (ISPOR US, 2018)
Real-world EQ-5D-5L utility values in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2018)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-reported severity of pain interference in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (ANNEM 2023)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Lower extremity + upper limb disability in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A
Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray AJ, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Sénéchal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 2022 24(1): 7–17 )
Depression in patients with Charcot-Marie-Tooth
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Patient-reported symptom burden of Charcot-Marie-Tooth_PNS_2022
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Work impacts in patients with Charcot-Marie-Tooth_PNS_2022
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Disease burden and quality of life in patients with Charcot-Marie-Tooth disease type 1A, 2, 4 and X in Europe and the US measured using a digital app
Ziemssen T, Thomas FP, Sivera R, Saporta M, Genovese F, Gray A, Hollett C, Moore A, Rego M, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Larkin M, Ouyang C, Quinn C (Muscle & Nerve, 62: S69-S70)
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study
Thomas FP, Saporta M, Attarian S, Sevilla T, Mascaró RS, Fabrizi GM, Genovese F, Gray A, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Monteiro K, Paoli X, Llewellyn S, Larkin M, Boutalbi Y (Neurodegenerative Disease Management, 2021; 11(1): 21-33)
Patient-reported symptom burden of Charcot-Marie-Tooth disease Type 1a in Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343)
Patient-reported burden of Charcot-Marie-Tooth disease Type 1a In Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343-344)
Productivity losses in people with Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world evidence study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Healthcare resource use in Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Demographics and treatment patterns for Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Diversity in the Charcot–Marie–Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment of the Charcot-Marie-Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Monteiro K, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment patterns for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Care standards for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Diversity in the Charcot-Marie-Tooth disease population in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Digitally assessed real-world diversity in people with Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Digitally assessed patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Development of a digital app and innovative recruitment for an international real-world observational study in Charcot-Marie-Tooth disease
Ziemssen T, Attarian S, Thomas FP, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Kousoulakou H, Larkin M (ISPOR EU, 2018)
Patient-reported impact of adult-onset ALSP
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Caregiving burden among caregivers of people with myasthenia gravis
Dewilde S, Tollenaar NH, Boulanger P, Archer A, Pardo R, Cortés-Vicente E, Mantegazza R, Vanoli F, Lehnerer S, Pawlitzki M, Heinrich M, De Ruyck F, Phillips G, Paci S (Orphanet Journal of Rare Diseases, 2025)
Harnessing digital measures of physical function: findings from a targeted literature review
Aggio D, Ringland C, Amini F, Bowmar E, Llewellyn S (ISPOR, 2025)
A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry
Amini F, Lawrence J, Vincent S, Penduka T, Hall G, Fellows A, Larkin M, Scowcroft A, Beau Lejdstrom R, Savic N, Jacob S, Habib A, Saccà F, Hayes A (AAN, 2025 and MGFA International, 2025)
The interplay between CASA-Q domains: insights from a real-world study investigating the effects of extended-release guaifenesin in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Fellows A, Llewellyn S (ACC, 2025)
Adjunctive long-term Mucinex® use leading to improvement in stable chronic bronchitis and decreased health care resource utilization – a case report
Divel C, Borecka O, Llewellyn S, Patel P, Shea T, Adeleke M, Spangenthal S (ATS, 2025)
Modified Delphi panel on advanced ALK-positive NSCLC patient profiling, treatment landscape, and clinical guidelines in the UK
Patel A, Tsang C, Baig LE, Llewellyn S, Baijal S (BTOG, 2025)
Caregiver-reported quality of life of patients with MCT8 deficiency: Results from a cross-sectional survey
Ofori A, Larkin M, Georges N (ISPOR Europe, 2024)
Quality of Life Impact of Caregiving for Patients with MCT8 Deficiency: Results from a Cross-Sectional Survey
Ofori A, Larkin M, Georges N (ISPOR EU, 2024)
Quality of patient engagement activities in health economics and outcomes research: insights from the ISPOR community
Llewellyn S, Qiao N, Masurkar P, Botto-van Bemden A, Roydhouse J, Perfetto E (Value & Outcomes Spotlight, 10(6):41-45)
Development of two conceptual models to describe the patient and caregiver reported impacts of living with X-linked myotubular myopathy (XLMTM)
Borecka O, Lawrence J, Llewellyn S (ISOQOL, 2024)
Leveraging SMS for patient-reported data collection
Llewellyn S, Amini F (ISOQOL, 2024)
Duchenne muscular dystrophy (DMD) patient vignettes development methodology
Merla V, Posner N, Borecka O, Vincent S (EAN, 2024)
Real world effectiveness of guaifenesin ER in tackling mucus hypersecretion in stable chronic bronchitis
Spangenthal S, Llewellyn S, Borecka O, Pollack C, Adeleke M, Kulasekaran A, Birring S, Mazzone S, Shea T (The Thirteenth London International Cough Symposium, 2024)
Patient-reported outcome measures used in interventional studies of irritable bowel syndrome: a targeted review
Lawrence J, Boxell E (National PROMS UK, 2024)
Outcomes and interventions in clinical studies investigating pharmacological therapies for the treatment of transthyretin amyloidosis: a targeted review
Alsawady M, Borecka O (National PROMS UK, 2024)
Diagnosis Validation in Patient-Centered Research
Alsawady M, Lawrence J, Amini F, Vincent S (Value and Outcomes Spotlight, 2024)
Assessing adherence in real-world studies
Ofori A, Octavia Borecka O, Llewellyn S
Approaches and challenges in developing caregiver vignettes in rare, progressive, and fatal childhood conditions: learnings from vignette development in Duchenne muscular dystrophy (DMD)
Llewellyn S, Borecka O, Bottomley C, Richardson L, Biggane A (ISOQOL, 2023)
Caregiver burden in growth hormone deficiency: a targeted review
Amini F, Alsawady M, Borecka O, Bottomley C
(ISPOR EU, 2023)
“I’ve got a text!” Feedback from prospective participants on the use of SMS surveys in real-world studies
Lawrence J, Amini F, Monera-Penduka TG, Llewellyn S
(ISPOR EU, 2023)
Vitamin D as an adjunct therapy in the treatment of atopic dermatitis: a targeted review
Borecka O, Lawrence J, Llewellyn S (ISPOR EU, 2023)
A targeted review of pharmacological therapies for the treatment of chronic spontaneous urticaria
Lawrence J, Borecka O, Alsawady M, Sauca M, Kallmes K, Llewellyn S (ISPOR EU, 2023)
Adverse events associated with clinical studies
Alsawady M, Borecka O & Llewellyn S (ISPOR US, 2023)
Patient feedback on the use of reward programs
Alsawady M, Amini F, Vincent S, Llewellyn S (ISPOR US, 2023)
Effectiveness of prostate cancer screening
Llewellyn S (ISPOR US, 2023)
Engagement statistics in real-world studies
Day L, Lau J, Quinn C (ISPOR EU, 2022)
Adherence rates in real-world studies
Borecka O, Ofori A & Llewellyn S (ISPOR EU, 2022)
Digital diagnosis validation feature
Vincent S, Borecka O, Llewellyn S & Bottomley C (ISPOR EU, 2022)
White paper: Patient-centric real-world evidence
The power of technology in real-world evidence generation
In today’s healthcare market, patient-centric evidence is crucial for product differentiation and success.
Diagnosis validation in patient-centered research
Llewellyn S, Bottomley C, Borecka O (National PROMs Annual Research Conference, 2022)
Patient-centricity with digital real-world evidence
When it comes to drug discovery there is a wealth of insight to be gained from the patient community. Involving patients in the design and development of real-world evidence studies, which in turn complement traditional clinical trials by demonstrating patients’ lived experience of the condition and its treatments, can be a powerful means of emphasizing patient-centricity in drug development. In this article, the authors explore the technicalities of engaging patients in real-world evidence studies.
Building a bridge between patient and pharma
To what extent does the scientific relevance of a healthcare research question parallel its relevance from the patient’s perspective? In this article, we use a live example of a real-world study investigating the experiences of individuals living with Charcot-Marie-Tooth disease (CMT) to demonstrate the wide-reaching value of patient involvement in study design and development.
Reimbursement of digital therapeutics
A comparison of the reimbursement of digital therapeutics in France, Germany, and the UK. Presented at ISPOR EU 2021.
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (Global Genes RARE Patient Advocacy Summit, 2022)
Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)
This manuscript was published in BMJ Open in May 2023. It discusses the patient-reported impact of myasthenia gravis in the real world using findings from a digital observational survey-based study (MyRealWorld MG).See here for more of our publications.Author Author...
Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG
Dewilde S, Philips G, Paci S, Beauchamp J, Chiroli S, Quinn C, Day L, Larkin M, Palace J, Berrih-Aknin S, Claeys K, Muppidi S, Mantegazza R, Saccà F, Meisel A, Bassez G, Murai H, Janssen MF (BMJ Open, 2022)
WHITE PAPER: Unleashing the potential of real-world evidence in rare diseases
Murthy A, Mantegazza R, Larkin M, Friconneau M, Annemans L, Van Damme B, Sokol T, Emond S (2022)
Digital data collection to measure the impact of myasthenia gravis on patients’ utility values in the real world
Dewilde S, Kousoulakou H, Janssen M, Claeys K, Friconneau M, Jacob S, Meisel A, Day L, Quinn C, Larkin M, Leighton T, Phillips G, Paci S (Value in Health, 25(1): S246)
Real-world patient-reported impact of myasthenia gravis: initial data from the MyRealWorld™ MG study
This poster was presented in October 2021 at the AANEM annual meeting. It shares the initial data from the MyRealWorld™ MG study about the impact of myasthenia gravis on the daily lives of patients.See here for more of our publications on myasthenia gravis and other...
Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
Berrih-Aknin S, Claeys K, Law N, Mantegazza R, Murai H, Sacca F, Dewilde S, Janssen M, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (BMJ Open 2021;11:e048198. doi: 10.1136/bmjopen-2020-048198)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Assouly P, Berrih-Aknin S, Claeys K, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J (Muscle & Nerve, 62: S65-S66)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Palace J, Assouly P, Claeys KG, Saccà F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (ABN, 2020)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Meisel A, Assouly P, Berrih-Aknin S, Claeys KG, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (DGN, 2020)
What role does caregiver burden play in health technology assessment bodies’ appraisals of highly specialized technologies?
Ofori A, Llewellyn S, Bottomley C, Kousoulakou H (ISPOR EU, 2020)
Sickle cell disease patient registries are not collecting enough data
Llewellyn S, Doe A, Åkesson C, Kousoulakou H, Quinn C, Larkin M (ISPOR US, 2020)
Complexities in localizing participant profile survey questions in real-world studies for smartphone apps
Kretz S, Richards A (Quality of Life Research, 29: S135)
The clinical and cost-effectiveness of supplemental parenteral nutrition in oncology
Webb N, Fricke J, Hancock E, Trueman D, Ghosh S, Winstone J, Miners A, Shepelev J, Valle JW (ESMO Open. 2020 Jun 1;5(3):e000709)
Challenges with using paper format legacy Patient Reported Outcome Measures (PROMs) in digital real-world evidence (RWE) studies for apps
Kretz S, Spinage C, Bottomley C (Quality of Life Research, 29: S137)
Innovative partnerships with patients and patient advocacy groups in digital observational rare disease studies.
Llewellyn S,Bagshaw E,Williams H,Larkin M (RARE Patient Advocacy Summit; 2019 Sep 18-20; San Diego, CA, US)
Cystic fibrosis
What is the consistency of response to rimegepant at a group level in the acute treatment of migraine in UK adult patients? Protocol for a real-world, patient-centred study
O’Neil G, Abraham L, Pawinski R, Nakajima K, Bagshaw E, Fellows A, Llewellyn S, Lambru G (EHC, 2025)
Assessment of stable chronic bronchitis improvement with adjunctive long-term Mucinex® use via the cough and sputum assessment questionnaire (CASA-Q)
Divel C, Borecka O, Llewellyn S, Spangenthal S (CHEST, 2025)
Adjunctive long-term use of Mucinex® leading to improvement in stable chronic bronchitis and patient’s quality of life: A case report.
Spangenthal S, Divel C, Borecka O, Llewellyn S (Medical Reports, 2025)
Real-world use of Vitaccess Real™ platform to assess quality of life impact with long-term use of Mucinex® in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Llewellyn S (ISOQOL, 2025)
Multiple myeloma caregiver costs and disabilities data for economic modelling and HTA submissions
Alsawady M, Tarnowska R, Kudlac A, Vincent S, Melrose D, Lied-Lied A (ISPOR Europe, 2025)
A targeted review of caregiver experiences of CAR-T therapy in ambulatory settings
Ringland C, Bagshaw E, Llewellyn S, Pugh G (ISPOR Europe, 2025)
Extrapulmonary disease burden and impact of cystic fibrosis (CF) on productivity in people with CF (pwCF) aged >12 years not treated with CF transmembrane conductance regulator modulators (CFTRm): interim analysis (IA) of the HUBBLE study
Elborn S, Mainz J, Abbott J, Carr S, Sole A, Costa S, Ganapathy V, Arteaga-Solis E, Liu J, Yuan J, Thorat T, Llewellyn S, Larkin M, De Iorio F, Kaplowitz H (ITS Annual Scientific Meeting, 2021)
Patient‑ and Caregiver‑Reported Burden of Transfusion‑Dependent β‑Thalassemia Measured Using a Digital Application
Paramore C, Levine L, Bagshaw E, Ouyang C, Kudlac A, Larkin M (Springer Nature’s, The Patient – Patient-Centered Outcomes Research, 2020)
Patient-reported burden of transfusion-dependent Beta-Thalassemia in the USA and UK measured using a digital app
Paramore C, Helfer J, Levine L, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Deprivation and access to care among patients with melanoma in the UK
Quinn C, Larkin M, Jacobs I, Kandola S, Smoyer K (ISPOR EU, 2022)
Using patient-reported data to estimate costs associated with melanoma
Javed M, Quinn C, Amini F, Boxell E (National PROMs Annual Research Conference, 2022)
Symptoms and side effects associated with melanoma treatment
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL 2021)
Contextualizing qualitative analysis with quantitative data on the symptoms and side effects associated with melanoma treatment in the real world – the value of patient registries
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL, 2021)
How do patients manage side effects during melanoma treatment? The importance of social support and doctor–patient communication
Ofori A, Hall K, Bottomley C, Larkin M, Heinrich M (The Christie School of Oncology, Melanoma Study Day, 2021)
Rewarding participants through charitable donations to Melanoma UK motivates recruitment and engagement: a bring your own device study example
Hall K, Ouyang C, Chilongo A, Llewellyn S, Quinn C, Larkin M (Quality of Life Research; 29: S91-S92)
Patient-centricity in action: using patient feedback to adapt the PRO-CTCAE instrument in a real-world study
Llewellyn S, Hall K, Camidge L, Bottomley C, Nixon A, Larkin M (Quality of Life Research, 29: S91)
Melanoma patient registries: a targeted review and comparison of datasets
Hall K, Llewellyn S, Bottomley C, Larkin M (Quality of Life Research, 29: S113)
Assessing the value of real-world evidence in melanoma in health technology assessment appraisals
Llewellyn S, Doe A, Quinn C (DIA Europe, 2020)
Targeted review of adverse events associated with treatments for stage 3 and 4 melanoma
Llewellyn S, Bagshaw E, Åkesson C, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Adverse events associated with immunotherapies used in the treatment of stage 3 and 4 melanoma
Llewellyn S, Åkesson C, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for melanoma in the UK measured using a digital ‘bring your own device’ platform
Larkin J, Cannon D, Nuttall G, Au L, Hunter N, Spain L, Turajlic S, Åkesson C, Llewellyn S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2019)
Real-world HRQL in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (PROMS Conference, 2018)
Taking the burden out of symptom reporting in patients with melanoma using a digital real-world evidence platform
Au L, Nuttall G, Cannon D, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Larkin J, Wiseman T (ISPOR US, 2018)
Real-world EQ-5D-5L utility values in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2018)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-reported severity of pain interference in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (ANNEM 2023)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Lower extremity + upper limb disability in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A
Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray AJ, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Sénéchal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 2022 24(1): 7–17 )
Depression in patients with Charcot-Marie-Tooth
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Patient-reported symptom burden of Charcot-Marie-Tooth_PNS_2022
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Work impacts in patients with Charcot-Marie-Tooth_PNS_2022
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Disease burden and quality of life in patients with Charcot-Marie-Tooth disease type 1A, 2, 4 and X in Europe and the US measured using a digital app
Ziemssen T, Thomas FP, Sivera R, Saporta M, Genovese F, Gray A, Hollett C, Moore A, Rego M, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Larkin M, Ouyang C, Quinn C (Muscle & Nerve, 62: S69-S70)
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study
Thomas FP, Saporta M, Attarian S, Sevilla T, Mascaró RS, Fabrizi GM, Genovese F, Gray A, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Monteiro K, Paoli X, Llewellyn S, Larkin M, Boutalbi Y (Neurodegenerative Disease Management, 2021; 11(1): 21-33)
Patient-reported symptom burden of Charcot-Marie-Tooth disease Type 1a in Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343)
Patient-reported burden of Charcot-Marie-Tooth disease Type 1a In Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343-344)
Productivity losses in people with Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world evidence study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Healthcare resource use in Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Demographics and treatment patterns for Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Diversity in the Charcot–Marie–Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment of the Charcot-Marie-Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Monteiro K, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment patterns for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Care standards for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Diversity in the Charcot-Marie-Tooth disease population in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Digitally assessed real-world diversity in people with Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Digitally assessed patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Development of a digital app and innovative recruitment for an international real-world observational study in Charcot-Marie-Tooth disease
Ziemssen T, Attarian S, Thomas FP, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Kousoulakou H, Larkin M (ISPOR EU, 2018)
Patient-reported impact of adult-onset ALSP
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Caregiving burden among caregivers of people with myasthenia gravis
Dewilde S, Tollenaar NH, Boulanger P, Archer A, Pardo R, Cortés-Vicente E, Mantegazza R, Vanoli F, Lehnerer S, Pawlitzki M, Heinrich M, De Ruyck F, Phillips G, Paci S (Orphanet Journal of Rare Diseases, 2025)
Harnessing digital measures of physical function: findings from a targeted literature review
Aggio D, Ringland C, Amini F, Bowmar E, Llewellyn S (ISPOR, 2025)
A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry
Amini F, Lawrence J, Vincent S, Penduka T, Hall G, Fellows A, Larkin M, Scowcroft A, Beau Lejdstrom R, Savic N, Jacob S, Habib A, Saccà F, Hayes A (AAN, 2025 and MGFA International, 2025)
The interplay between CASA-Q domains: insights from a real-world study investigating the effects of extended-release guaifenesin in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Fellows A, Llewellyn S (ACC, 2025)
Adjunctive long-term Mucinex® use leading to improvement in stable chronic bronchitis and decreased health care resource utilization – a case report
Divel C, Borecka O, Llewellyn S, Patel P, Shea T, Adeleke M, Spangenthal S (ATS, 2025)
Modified Delphi panel on advanced ALK-positive NSCLC patient profiling, treatment landscape, and clinical guidelines in the UK
Patel A, Tsang C, Baig LE, Llewellyn S, Baijal S (BTOG, 2025)
Caregiver-reported quality of life of patients with MCT8 deficiency: Results from a cross-sectional survey
Ofori A, Larkin M, Georges N (ISPOR Europe, 2024)
Quality of Life Impact of Caregiving for Patients with MCT8 Deficiency: Results from a Cross-Sectional Survey
Ofori A, Larkin M, Georges N (ISPOR EU, 2024)
Quality of patient engagement activities in health economics and outcomes research: insights from the ISPOR community
Llewellyn S, Qiao N, Masurkar P, Botto-van Bemden A, Roydhouse J, Perfetto E (Value & Outcomes Spotlight, 10(6):41-45)
Development of two conceptual models to describe the patient and caregiver reported impacts of living with X-linked myotubular myopathy (XLMTM)
Borecka O, Lawrence J, Llewellyn S (ISOQOL, 2024)
Leveraging SMS for patient-reported data collection
Llewellyn S, Amini F (ISOQOL, 2024)
Duchenne muscular dystrophy (DMD) patient vignettes development methodology
Merla V, Posner N, Borecka O, Vincent S (EAN, 2024)
Real world effectiveness of guaifenesin ER in tackling mucus hypersecretion in stable chronic bronchitis
Spangenthal S, Llewellyn S, Borecka O, Pollack C, Adeleke M, Kulasekaran A, Birring S, Mazzone S, Shea T (The Thirteenth London International Cough Symposium, 2024)
Patient-reported outcome measures used in interventional studies of irritable bowel syndrome: a targeted review
Lawrence J, Boxell E (National PROMS UK, 2024)
Outcomes and interventions in clinical studies investigating pharmacological therapies for the treatment of transthyretin amyloidosis: a targeted review
Alsawady M, Borecka O (National PROMS UK, 2024)
Diagnosis Validation in Patient-Centered Research
Alsawady M, Lawrence J, Amini F, Vincent S (Value and Outcomes Spotlight, 2024)
Assessing adherence in real-world studies
Ofori A, Octavia Borecka O, Llewellyn S
Approaches and challenges in developing caregiver vignettes in rare, progressive, and fatal childhood conditions: learnings from vignette development in Duchenne muscular dystrophy (DMD)
Llewellyn S, Borecka O, Bottomley C, Richardson L, Biggane A (ISOQOL, 2023)
Caregiver burden in growth hormone deficiency: a targeted review
Amini F, Alsawady M, Borecka O, Bottomley C
(ISPOR EU, 2023)
“I’ve got a text!” Feedback from prospective participants on the use of SMS surveys in real-world studies
Lawrence J, Amini F, Monera-Penduka TG, Llewellyn S
(ISPOR EU, 2023)
Vitamin D as an adjunct therapy in the treatment of atopic dermatitis: a targeted review
Borecka O, Lawrence J, Llewellyn S (ISPOR EU, 2023)
A targeted review of pharmacological therapies for the treatment of chronic spontaneous urticaria
Lawrence J, Borecka O, Alsawady M, Sauca M, Kallmes K, Llewellyn S (ISPOR EU, 2023)
Adverse events associated with clinical studies
Alsawady M, Borecka O & Llewellyn S (ISPOR US, 2023)
Patient feedback on the use of reward programs
Alsawady M, Amini F, Vincent S, Llewellyn S (ISPOR US, 2023)
Effectiveness of prostate cancer screening
Llewellyn S (ISPOR US, 2023)
Engagement statistics in real-world studies
Day L, Lau J, Quinn C (ISPOR EU, 2022)
Adherence rates in real-world studies
Borecka O, Ofori A & Llewellyn S (ISPOR EU, 2022)
Digital diagnosis validation feature
Vincent S, Borecka O, Llewellyn S & Bottomley C (ISPOR EU, 2022)
White paper: Patient-centric real-world evidence
The power of technology in real-world evidence generation
In today’s healthcare market, patient-centric evidence is crucial for product differentiation and success.
Diagnosis validation in patient-centered research
Llewellyn S, Bottomley C, Borecka O (National PROMs Annual Research Conference, 2022)
Patient-centricity with digital real-world evidence
When it comes to drug discovery there is a wealth of insight to be gained from the patient community. Involving patients in the design and development of real-world evidence studies, which in turn complement traditional clinical trials by demonstrating patients’ lived experience of the condition and its treatments, can be a powerful means of emphasizing patient-centricity in drug development. In this article, the authors explore the technicalities of engaging patients in real-world evidence studies.
Building a bridge between patient and pharma
To what extent does the scientific relevance of a healthcare research question parallel its relevance from the patient’s perspective? In this article, we use a live example of a real-world study investigating the experiences of individuals living with Charcot-Marie-Tooth disease (CMT) to demonstrate the wide-reaching value of patient involvement in study design and development.
Reimbursement of digital therapeutics
A comparison of the reimbursement of digital therapeutics in France, Germany, and the UK. Presented at ISPOR EU 2021.
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (Global Genes RARE Patient Advocacy Summit, 2022)
Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)
This manuscript was published in BMJ Open in May 2023. It discusses the patient-reported impact of myasthenia gravis in the real world using findings from a digital observational survey-based study (MyRealWorld MG).See here for more of our publications.Author Author...
Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG
Dewilde S, Philips G, Paci S, Beauchamp J, Chiroli S, Quinn C, Day L, Larkin M, Palace J, Berrih-Aknin S, Claeys K, Muppidi S, Mantegazza R, Saccà F, Meisel A, Bassez G, Murai H, Janssen MF (BMJ Open, 2022)
WHITE PAPER: Unleashing the potential of real-world evidence in rare diseases
Murthy A, Mantegazza R, Larkin M, Friconneau M, Annemans L, Van Damme B, Sokol T, Emond S (2022)
Digital data collection to measure the impact of myasthenia gravis on patients’ utility values in the real world
Dewilde S, Kousoulakou H, Janssen M, Claeys K, Friconneau M, Jacob S, Meisel A, Day L, Quinn C, Larkin M, Leighton T, Phillips G, Paci S (Value in Health, 25(1): S246)
Real-world patient-reported impact of myasthenia gravis: initial data from the MyRealWorld™ MG study
This poster was presented in October 2021 at the AANEM annual meeting. It shares the initial data from the MyRealWorld™ MG study about the impact of myasthenia gravis on the daily lives of patients.See here for more of our publications on myasthenia gravis and other...
Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
Berrih-Aknin S, Claeys K, Law N, Mantegazza R, Murai H, Sacca F, Dewilde S, Janssen M, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (BMJ Open 2021;11:e048198. doi: 10.1136/bmjopen-2020-048198)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Assouly P, Berrih-Aknin S, Claeys K, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J (Muscle & Nerve, 62: S65-S66)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Palace J, Assouly P, Claeys KG, Saccà F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (ABN, 2020)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Meisel A, Assouly P, Berrih-Aknin S, Claeys KG, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (DGN, 2020)
What role does caregiver burden play in health technology assessment bodies’ appraisals of highly specialized technologies?
Ofori A, Llewellyn S, Bottomley C, Kousoulakou H (ISPOR EU, 2020)
Sickle cell disease patient registries are not collecting enough data
Llewellyn S, Doe A, Åkesson C, Kousoulakou H, Quinn C, Larkin M (ISPOR US, 2020)
Complexities in localizing participant profile survey questions in real-world studies for smartphone apps
Kretz S, Richards A (Quality of Life Research, 29: S135)
The clinical and cost-effectiveness of supplemental parenteral nutrition in oncology
Webb N, Fricke J, Hancock E, Trueman D, Ghosh S, Winstone J, Miners A, Shepelev J, Valle JW (ESMO Open. 2020 Jun 1;5(3):e000709)
Challenges with using paper format legacy Patient Reported Outcome Measures (PROMs) in digital real-world evidence (RWE) studies for apps
Kretz S, Spinage C, Bottomley C (Quality of Life Research, 29: S137)
Innovative partnerships with patients and patient advocacy groups in digital observational rare disease studies.
Llewellyn S,Bagshaw E,Williams H,Larkin M (RARE Patient Advocacy Summit; 2019 Sep 18-20; San Diego, CA, US)
Beta-thalassemia
What is the consistency of response to rimegepant at a group level in the acute treatment of migraine in UK adult patients? Protocol for a real-world, patient-centred study
O’Neil G, Abraham L, Pawinski R, Nakajima K, Bagshaw E, Fellows A, Llewellyn S, Lambru G (EHC, 2025)
Assessment of stable chronic bronchitis improvement with adjunctive long-term Mucinex® use via the cough and sputum assessment questionnaire (CASA-Q)
Divel C, Borecka O, Llewellyn S, Spangenthal S (CHEST, 2025)
Adjunctive long-term use of Mucinex® leading to improvement in stable chronic bronchitis and patient’s quality of life: A case report.
Spangenthal S, Divel C, Borecka O, Llewellyn S (Medical Reports, 2025)
Real-world use of Vitaccess Real™ platform to assess quality of life impact with long-term use of Mucinex® in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Llewellyn S (ISOQOL, 2025)
Multiple myeloma caregiver costs and disabilities data for economic modelling and HTA submissions
Alsawady M, Tarnowska R, Kudlac A, Vincent S, Melrose D, Lied-Lied A (ISPOR Europe, 2025)
A targeted review of caregiver experiences of CAR-T therapy in ambulatory settings
Ringland C, Bagshaw E, Llewellyn S, Pugh G (ISPOR Europe, 2025)
Extrapulmonary disease burden and impact of cystic fibrosis (CF) on productivity in people with CF (pwCF) aged >12 years not treated with CF transmembrane conductance regulator modulators (CFTRm): interim analysis (IA) of the HUBBLE study
Elborn S, Mainz J, Abbott J, Carr S, Sole A, Costa S, Ganapathy V, Arteaga-Solis E, Liu J, Yuan J, Thorat T, Llewellyn S, Larkin M, De Iorio F, Kaplowitz H (ITS Annual Scientific Meeting, 2021)
Patient‑ and Caregiver‑Reported Burden of Transfusion‑Dependent β‑Thalassemia Measured Using a Digital Application
Paramore C, Levine L, Bagshaw E, Ouyang C, Kudlac A, Larkin M (Springer Nature’s, The Patient – Patient-Centered Outcomes Research, 2020)
Patient-reported burden of transfusion-dependent Beta-Thalassemia in the USA and UK measured using a digital app
Paramore C, Helfer J, Levine L, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Deprivation and access to care among patients with melanoma in the UK
Quinn C, Larkin M, Jacobs I, Kandola S, Smoyer K (ISPOR EU, 2022)
Using patient-reported data to estimate costs associated with melanoma
Javed M, Quinn C, Amini F, Boxell E (National PROMs Annual Research Conference, 2022)
Symptoms and side effects associated with melanoma treatment
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL 2021)
Contextualizing qualitative analysis with quantitative data on the symptoms and side effects associated with melanoma treatment in the real world – the value of patient registries
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL, 2021)
How do patients manage side effects during melanoma treatment? The importance of social support and doctor–patient communication
Ofori A, Hall K, Bottomley C, Larkin M, Heinrich M (The Christie School of Oncology, Melanoma Study Day, 2021)
Rewarding participants through charitable donations to Melanoma UK motivates recruitment and engagement: a bring your own device study example
Hall K, Ouyang C, Chilongo A, Llewellyn S, Quinn C, Larkin M (Quality of Life Research; 29: S91-S92)
Patient-centricity in action: using patient feedback to adapt the PRO-CTCAE instrument in a real-world study
Llewellyn S, Hall K, Camidge L, Bottomley C, Nixon A, Larkin M (Quality of Life Research, 29: S91)
Melanoma patient registries: a targeted review and comparison of datasets
Hall K, Llewellyn S, Bottomley C, Larkin M (Quality of Life Research, 29: S113)
Assessing the value of real-world evidence in melanoma in health technology assessment appraisals
Llewellyn S, Doe A, Quinn C (DIA Europe, 2020)
Targeted review of adverse events associated with treatments for stage 3 and 4 melanoma
Llewellyn S, Bagshaw E, Åkesson C, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Adverse events associated with immunotherapies used in the treatment of stage 3 and 4 melanoma
Llewellyn S, Åkesson C, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for melanoma in the UK measured using a digital ‘bring your own device’ platform
Larkin J, Cannon D, Nuttall G, Au L, Hunter N, Spain L, Turajlic S, Åkesson C, Llewellyn S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2019)
Real-world HRQL in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (PROMS Conference, 2018)
Taking the burden out of symptom reporting in patients with melanoma using a digital real-world evidence platform
Au L, Nuttall G, Cannon D, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Larkin J, Wiseman T (ISPOR US, 2018)
Real-world EQ-5D-5L utility values in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2018)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-reported severity of pain interference in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (ANNEM 2023)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Lower extremity + upper limb disability in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A
Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray AJ, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Sénéchal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 2022 24(1): 7–17 )
Depression in patients with Charcot-Marie-Tooth
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Patient-reported symptom burden of Charcot-Marie-Tooth_PNS_2022
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Work impacts in patients with Charcot-Marie-Tooth_PNS_2022
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Disease burden and quality of life in patients with Charcot-Marie-Tooth disease type 1A, 2, 4 and X in Europe and the US measured using a digital app
Ziemssen T, Thomas FP, Sivera R, Saporta M, Genovese F, Gray A, Hollett C, Moore A, Rego M, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Larkin M, Ouyang C, Quinn C (Muscle & Nerve, 62: S69-S70)
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study
Thomas FP, Saporta M, Attarian S, Sevilla T, Mascaró RS, Fabrizi GM, Genovese F, Gray A, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Monteiro K, Paoli X, Llewellyn S, Larkin M, Boutalbi Y (Neurodegenerative Disease Management, 2021; 11(1): 21-33)
Patient-reported symptom burden of Charcot-Marie-Tooth disease Type 1a in Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343)
Patient-reported burden of Charcot-Marie-Tooth disease Type 1a In Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343-344)
Productivity losses in people with Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world evidence study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Healthcare resource use in Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Demographics and treatment patterns for Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Diversity in the Charcot–Marie–Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment of the Charcot-Marie-Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Monteiro K, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment patterns for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Care standards for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Diversity in the Charcot-Marie-Tooth disease population in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Digitally assessed real-world diversity in people with Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Digitally assessed patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Development of a digital app and innovative recruitment for an international real-world observational study in Charcot-Marie-Tooth disease
Ziemssen T, Attarian S, Thomas FP, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Kousoulakou H, Larkin M (ISPOR EU, 2018)
Patient-reported impact of adult-onset ALSP
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Caregiving burden among caregivers of people with myasthenia gravis
Dewilde S, Tollenaar NH, Boulanger P, Archer A, Pardo R, Cortés-Vicente E, Mantegazza R, Vanoli F, Lehnerer S, Pawlitzki M, Heinrich M, De Ruyck F, Phillips G, Paci S (Orphanet Journal of Rare Diseases, 2025)
Harnessing digital measures of physical function: findings from a targeted literature review
Aggio D, Ringland C, Amini F, Bowmar E, Llewellyn S (ISPOR, 2025)
A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry
Amini F, Lawrence J, Vincent S, Penduka T, Hall G, Fellows A, Larkin M, Scowcroft A, Beau Lejdstrom R, Savic N, Jacob S, Habib A, Saccà F, Hayes A (AAN, 2025 and MGFA International, 2025)
The interplay between CASA-Q domains: insights from a real-world study investigating the effects of extended-release guaifenesin in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Fellows A, Llewellyn S (ACC, 2025)
Adjunctive long-term Mucinex® use leading to improvement in stable chronic bronchitis and decreased health care resource utilization – a case report
Divel C, Borecka O, Llewellyn S, Patel P, Shea T, Adeleke M, Spangenthal S (ATS, 2025)
Modified Delphi panel on advanced ALK-positive NSCLC patient profiling, treatment landscape, and clinical guidelines in the UK
Patel A, Tsang C, Baig LE, Llewellyn S, Baijal S (BTOG, 2025)
Caregiver-reported quality of life of patients with MCT8 deficiency: Results from a cross-sectional survey
Ofori A, Larkin M, Georges N (ISPOR Europe, 2024)
Quality of Life Impact of Caregiving for Patients with MCT8 Deficiency: Results from a Cross-Sectional Survey
Ofori A, Larkin M, Georges N (ISPOR EU, 2024)
Quality of patient engagement activities in health economics and outcomes research: insights from the ISPOR community
Llewellyn S, Qiao N, Masurkar P, Botto-van Bemden A, Roydhouse J, Perfetto E (Value & Outcomes Spotlight, 10(6):41-45)
Development of two conceptual models to describe the patient and caregiver reported impacts of living with X-linked myotubular myopathy (XLMTM)
Borecka O, Lawrence J, Llewellyn S (ISOQOL, 2024)
Leveraging SMS for patient-reported data collection
Llewellyn S, Amini F (ISOQOL, 2024)
Duchenne muscular dystrophy (DMD) patient vignettes development methodology
Merla V, Posner N, Borecka O, Vincent S (EAN, 2024)
Real world effectiveness of guaifenesin ER in tackling mucus hypersecretion in stable chronic bronchitis
Spangenthal S, Llewellyn S, Borecka O, Pollack C, Adeleke M, Kulasekaran A, Birring S, Mazzone S, Shea T (The Thirteenth London International Cough Symposium, 2024)
Patient-reported outcome measures used in interventional studies of irritable bowel syndrome: a targeted review
Lawrence J, Boxell E (National PROMS UK, 2024)
Outcomes and interventions in clinical studies investigating pharmacological therapies for the treatment of transthyretin amyloidosis: a targeted review
Alsawady M, Borecka O (National PROMS UK, 2024)
Diagnosis Validation in Patient-Centered Research
Alsawady M, Lawrence J, Amini F, Vincent S (Value and Outcomes Spotlight, 2024)
Assessing adherence in real-world studies
Ofori A, Octavia Borecka O, Llewellyn S
Approaches and challenges in developing caregiver vignettes in rare, progressive, and fatal childhood conditions: learnings from vignette development in Duchenne muscular dystrophy (DMD)
Llewellyn S, Borecka O, Bottomley C, Richardson L, Biggane A (ISOQOL, 2023)
Caregiver burden in growth hormone deficiency: a targeted review
Amini F, Alsawady M, Borecka O, Bottomley C
(ISPOR EU, 2023)
“I’ve got a text!” Feedback from prospective participants on the use of SMS surveys in real-world studies
Lawrence J, Amini F, Monera-Penduka TG, Llewellyn S
(ISPOR EU, 2023)
Vitamin D as an adjunct therapy in the treatment of atopic dermatitis: a targeted review
Borecka O, Lawrence J, Llewellyn S (ISPOR EU, 2023)
A targeted review of pharmacological therapies for the treatment of chronic spontaneous urticaria
Lawrence J, Borecka O, Alsawady M, Sauca M, Kallmes K, Llewellyn S (ISPOR EU, 2023)
Adverse events associated with clinical studies
Alsawady M, Borecka O & Llewellyn S (ISPOR US, 2023)
Patient feedback on the use of reward programs
Alsawady M, Amini F, Vincent S, Llewellyn S (ISPOR US, 2023)
Effectiveness of prostate cancer screening
Llewellyn S (ISPOR US, 2023)
Engagement statistics in real-world studies
Day L, Lau J, Quinn C (ISPOR EU, 2022)
Adherence rates in real-world studies
Borecka O, Ofori A & Llewellyn S (ISPOR EU, 2022)
Digital diagnosis validation feature
Vincent S, Borecka O, Llewellyn S & Bottomley C (ISPOR EU, 2022)
White paper: Patient-centric real-world evidence
The power of technology in real-world evidence generation
In today’s healthcare market, patient-centric evidence is crucial for product differentiation and success.
Diagnosis validation in patient-centered research
Llewellyn S, Bottomley C, Borecka O (National PROMs Annual Research Conference, 2022)
Patient-centricity with digital real-world evidence
When it comes to drug discovery there is a wealth of insight to be gained from the patient community. Involving patients in the design and development of real-world evidence studies, which in turn complement traditional clinical trials by demonstrating patients’ lived experience of the condition and its treatments, can be a powerful means of emphasizing patient-centricity in drug development. In this article, the authors explore the technicalities of engaging patients in real-world evidence studies.
Building a bridge between patient and pharma
To what extent does the scientific relevance of a healthcare research question parallel its relevance from the patient’s perspective? In this article, we use a live example of a real-world study investigating the experiences of individuals living with Charcot-Marie-Tooth disease (CMT) to demonstrate the wide-reaching value of patient involvement in study design and development.
Reimbursement of digital therapeutics
A comparison of the reimbursement of digital therapeutics in France, Germany, and the UK. Presented at ISPOR EU 2021.
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (Global Genes RARE Patient Advocacy Summit, 2022)
Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)
This manuscript was published in BMJ Open in May 2023. It discusses the patient-reported impact of myasthenia gravis in the real world using findings from a digital observational survey-based study (MyRealWorld MG).See here for more of our publications.Author Author...
Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG
Dewilde S, Philips G, Paci S, Beauchamp J, Chiroli S, Quinn C, Day L, Larkin M, Palace J, Berrih-Aknin S, Claeys K, Muppidi S, Mantegazza R, Saccà F, Meisel A, Bassez G, Murai H, Janssen MF (BMJ Open, 2022)
WHITE PAPER: Unleashing the potential of real-world evidence in rare diseases
Murthy A, Mantegazza R, Larkin M, Friconneau M, Annemans L, Van Damme B, Sokol T, Emond S (2022)
Digital data collection to measure the impact of myasthenia gravis on patients’ utility values in the real world
Dewilde S, Kousoulakou H, Janssen M, Claeys K, Friconneau M, Jacob S, Meisel A, Day L, Quinn C, Larkin M, Leighton T, Phillips G, Paci S (Value in Health, 25(1): S246)
Real-world patient-reported impact of myasthenia gravis: initial data from the MyRealWorld™ MG study
This poster was presented in October 2021 at the AANEM annual meeting. It shares the initial data from the MyRealWorld™ MG study about the impact of myasthenia gravis on the daily lives of patients.See here for more of our publications on myasthenia gravis and other...
Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
Berrih-Aknin S, Claeys K, Law N, Mantegazza R, Murai H, Sacca F, Dewilde S, Janssen M, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (BMJ Open 2021;11:e048198. doi: 10.1136/bmjopen-2020-048198)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Assouly P, Berrih-Aknin S, Claeys K, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J (Muscle & Nerve, 62: S65-S66)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Palace J, Assouly P, Claeys KG, Saccà F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (ABN, 2020)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Meisel A, Assouly P, Berrih-Aknin S, Claeys KG, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (DGN, 2020)
What role does caregiver burden play in health technology assessment bodies’ appraisals of highly specialized technologies?
Ofori A, Llewellyn S, Bottomley C, Kousoulakou H (ISPOR EU, 2020)
Sickle cell disease patient registries are not collecting enough data
Llewellyn S, Doe A, Åkesson C, Kousoulakou H, Quinn C, Larkin M (ISPOR US, 2020)
Complexities in localizing participant profile survey questions in real-world studies for smartphone apps
Kretz S, Richards A (Quality of Life Research, 29: S135)
The clinical and cost-effectiveness of supplemental parenteral nutrition in oncology
Webb N, Fricke J, Hancock E, Trueman D, Ghosh S, Winstone J, Miners A, Shepelev J, Valle JW (ESMO Open. 2020 Jun 1;5(3):e000709)
Challenges with using paper format legacy Patient Reported Outcome Measures (PROMs) in digital real-world evidence (RWE) studies for apps
Kretz S, Spinage C, Bottomley C (Quality of Life Research, 29: S137)
Innovative partnerships with patients and patient advocacy groups in digital observational rare disease studies.
Llewellyn S,Bagshaw E,Williams H,Larkin M (RARE Patient Advocacy Summit; 2019 Sep 18-20; San Diego, CA, US)
Charcot-Marie-Tooth disease
What is the consistency of response to rimegepant at a group level in the acute treatment of migraine in UK adult patients? Protocol for a real-world, patient-centred study
O’Neil G, Abraham L, Pawinski R, Nakajima K, Bagshaw E, Fellows A, Llewellyn S, Lambru G (EHC, 2025)
Assessment of stable chronic bronchitis improvement with adjunctive long-term Mucinex® use via the cough and sputum assessment questionnaire (CASA-Q)
Divel C, Borecka O, Llewellyn S, Spangenthal S (CHEST, 2025)
Adjunctive long-term use of Mucinex® leading to improvement in stable chronic bronchitis and patient’s quality of life: A case report.
Spangenthal S, Divel C, Borecka O, Llewellyn S (Medical Reports, 2025)
Real-world use of Vitaccess Real™ platform to assess quality of life impact with long-term use of Mucinex® in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Llewellyn S (ISOQOL, 2025)
Multiple myeloma caregiver costs and disabilities data for economic modelling and HTA submissions
Alsawady M, Tarnowska R, Kudlac A, Vincent S, Melrose D, Lied-Lied A (ISPOR Europe, 2025)
A targeted review of caregiver experiences of CAR-T therapy in ambulatory settings
Ringland C, Bagshaw E, Llewellyn S, Pugh G (ISPOR Europe, 2025)
Extrapulmonary disease burden and impact of cystic fibrosis (CF) on productivity in people with CF (pwCF) aged >12 years not treated with CF transmembrane conductance regulator modulators (CFTRm): interim analysis (IA) of the HUBBLE study
Elborn S, Mainz J, Abbott J, Carr S, Sole A, Costa S, Ganapathy V, Arteaga-Solis E, Liu J, Yuan J, Thorat T, Llewellyn S, Larkin M, De Iorio F, Kaplowitz H (ITS Annual Scientific Meeting, 2021)
Patient‑ and Caregiver‑Reported Burden of Transfusion‑Dependent β‑Thalassemia Measured Using a Digital Application
Paramore C, Levine L, Bagshaw E, Ouyang C, Kudlac A, Larkin M (Springer Nature’s, The Patient – Patient-Centered Outcomes Research, 2020)
Patient-reported burden of transfusion-dependent Beta-Thalassemia in the USA and UK measured using a digital app
Paramore C, Helfer J, Levine L, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Deprivation and access to care among patients with melanoma in the UK
Quinn C, Larkin M, Jacobs I, Kandola S, Smoyer K (ISPOR EU, 2022)
Using patient-reported data to estimate costs associated with melanoma
Javed M, Quinn C, Amini F, Boxell E (National PROMs Annual Research Conference, 2022)
Symptoms and side effects associated with melanoma treatment
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL 2021)
Contextualizing qualitative analysis with quantitative data on the symptoms and side effects associated with melanoma treatment in the real world – the value of patient registries
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL, 2021)
How do patients manage side effects during melanoma treatment? The importance of social support and doctor–patient communication
Ofori A, Hall K, Bottomley C, Larkin M, Heinrich M (The Christie School of Oncology, Melanoma Study Day, 2021)
Rewarding participants through charitable donations to Melanoma UK motivates recruitment and engagement: a bring your own device study example
Hall K, Ouyang C, Chilongo A, Llewellyn S, Quinn C, Larkin M (Quality of Life Research; 29: S91-S92)
Patient-centricity in action: using patient feedback to adapt the PRO-CTCAE instrument in a real-world study
Llewellyn S, Hall K, Camidge L, Bottomley C, Nixon A, Larkin M (Quality of Life Research, 29: S91)
Melanoma patient registries: a targeted review and comparison of datasets
Hall K, Llewellyn S, Bottomley C, Larkin M (Quality of Life Research, 29: S113)
Assessing the value of real-world evidence in melanoma in health technology assessment appraisals
Llewellyn S, Doe A, Quinn C (DIA Europe, 2020)
Targeted review of adverse events associated with treatments for stage 3 and 4 melanoma
Llewellyn S, Bagshaw E, Åkesson C, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Adverse events associated with immunotherapies used in the treatment of stage 3 and 4 melanoma
Llewellyn S, Åkesson C, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for melanoma in the UK measured using a digital ‘bring your own device’ platform
Larkin J, Cannon D, Nuttall G, Au L, Hunter N, Spain L, Turajlic S, Åkesson C, Llewellyn S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2019)
Real-world HRQL in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (PROMS Conference, 2018)
Taking the burden out of symptom reporting in patients with melanoma using a digital real-world evidence platform
Au L, Nuttall G, Cannon D, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Larkin J, Wiseman T (ISPOR US, 2018)
Real-world EQ-5D-5L utility values in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2018)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-reported severity of pain interference in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (ANNEM 2023)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Lower extremity + upper limb disability in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A
Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray AJ, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Sénéchal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 2022 24(1): 7–17 )
Depression in patients with Charcot-Marie-Tooth
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Patient-reported symptom burden of Charcot-Marie-Tooth_PNS_2022
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Work impacts in patients with Charcot-Marie-Tooth_PNS_2022
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Disease burden and quality of life in patients with Charcot-Marie-Tooth disease type 1A, 2, 4 and X in Europe and the US measured using a digital app
Ziemssen T, Thomas FP, Sivera R, Saporta M, Genovese F, Gray A, Hollett C, Moore A, Rego M, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Larkin M, Ouyang C, Quinn C (Muscle & Nerve, 62: S69-S70)
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study
Thomas FP, Saporta M, Attarian S, Sevilla T, Mascaró RS, Fabrizi GM, Genovese F, Gray A, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Monteiro K, Paoli X, Llewellyn S, Larkin M, Boutalbi Y (Neurodegenerative Disease Management, 2021; 11(1): 21-33)
Patient-reported symptom burden of Charcot-Marie-Tooth disease Type 1a in Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343)
Patient-reported burden of Charcot-Marie-Tooth disease Type 1a In Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343-344)
Productivity losses in people with Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world evidence study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Healthcare resource use in Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Demographics and treatment patterns for Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Diversity in the Charcot–Marie–Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment of the Charcot-Marie-Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Monteiro K, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment patterns for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Care standards for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Diversity in the Charcot-Marie-Tooth disease population in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Digitally assessed real-world diversity in people with Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Digitally assessed patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Development of a digital app and innovative recruitment for an international real-world observational study in Charcot-Marie-Tooth disease
Ziemssen T, Attarian S, Thomas FP, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Kousoulakou H, Larkin M (ISPOR EU, 2018)
Patient-reported impact of adult-onset ALSP
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Caregiving burden among caregivers of people with myasthenia gravis
Dewilde S, Tollenaar NH, Boulanger P, Archer A, Pardo R, Cortés-Vicente E, Mantegazza R, Vanoli F, Lehnerer S, Pawlitzki M, Heinrich M, De Ruyck F, Phillips G, Paci S (Orphanet Journal of Rare Diseases, 2025)
Harnessing digital measures of physical function: findings from a targeted literature review
Aggio D, Ringland C, Amini F, Bowmar E, Llewellyn S (ISPOR, 2025)
A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry
Amini F, Lawrence J, Vincent S, Penduka T, Hall G, Fellows A, Larkin M, Scowcroft A, Beau Lejdstrom R, Savic N, Jacob S, Habib A, Saccà F, Hayes A (AAN, 2025 and MGFA International, 2025)
The interplay between CASA-Q domains: insights from a real-world study investigating the effects of extended-release guaifenesin in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Fellows A, Llewellyn S (ACC, 2025)
Adjunctive long-term Mucinex® use leading to improvement in stable chronic bronchitis and decreased health care resource utilization – a case report
Divel C, Borecka O, Llewellyn S, Patel P, Shea T, Adeleke M, Spangenthal S (ATS, 2025)
Modified Delphi panel on advanced ALK-positive NSCLC patient profiling, treatment landscape, and clinical guidelines in the UK
Patel A, Tsang C, Baig LE, Llewellyn S, Baijal S (BTOG, 2025)
Caregiver-reported quality of life of patients with MCT8 deficiency: Results from a cross-sectional survey
Ofori A, Larkin M, Georges N (ISPOR Europe, 2024)
Quality of Life Impact of Caregiving for Patients with MCT8 Deficiency: Results from a Cross-Sectional Survey
Ofori A, Larkin M, Georges N (ISPOR EU, 2024)
Quality of patient engagement activities in health economics and outcomes research: insights from the ISPOR community
Llewellyn S, Qiao N, Masurkar P, Botto-van Bemden A, Roydhouse J, Perfetto E (Value & Outcomes Spotlight, 10(6):41-45)
Development of two conceptual models to describe the patient and caregiver reported impacts of living with X-linked myotubular myopathy (XLMTM)
Borecka O, Lawrence J, Llewellyn S (ISOQOL, 2024)
Leveraging SMS for patient-reported data collection
Llewellyn S, Amini F (ISOQOL, 2024)
Duchenne muscular dystrophy (DMD) patient vignettes development methodology
Merla V, Posner N, Borecka O, Vincent S (EAN, 2024)
Real world effectiveness of guaifenesin ER in tackling mucus hypersecretion in stable chronic bronchitis
Spangenthal S, Llewellyn S, Borecka O, Pollack C, Adeleke M, Kulasekaran A, Birring S, Mazzone S, Shea T (The Thirteenth London International Cough Symposium, 2024)
Patient-reported outcome measures used in interventional studies of irritable bowel syndrome: a targeted review
Lawrence J, Boxell E (National PROMS UK, 2024)
Outcomes and interventions in clinical studies investigating pharmacological therapies for the treatment of transthyretin amyloidosis: a targeted review
Alsawady M, Borecka O (National PROMS UK, 2024)
Diagnosis Validation in Patient-Centered Research
Alsawady M, Lawrence J, Amini F, Vincent S (Value and Outcomes Spotlight, 2024)
Assessing adherence in real-world studies
Ofori A, Octavia Borecka O, Llewellyn S
Approaches and challenges in developing caregiver vignettes in rare, progressive, and fatal childhood conditions: learnings from vignette development in Duchenne muscular dystrophy (DMD)
Llewellyn S, Borecka O, Bottomley C, Richardson L, Biggane A (ISOQOL, 2023)
Caregiver burden in growth hormone deficiency: a targeted review
Amini F, Alsawady M, Borecka O, Bottomley C
(ISPOR EU, 2023)
“I’ve got a text!” Feedback from prospective participants on the use of SMS surveys in real-world studies
Lawrence J, Amini F, Monera-Penduka TG, Llewellyn S
(ISPOR EU, 2023)
Vitamin D as an adjunct therapy in the treatment of atopic dermatitis: a targeted review
Borecka O, Lawrence J, Llewellyn S (ISPOR EU, 2023)
A targeted review of pharmacological therapies for the treatment of chronic spontaneous urticaria
Lawrence J, Borecka O, Alsawady M, Sauca M, Kallmes K, Llewellyn S (ISPOR EU, 2023)
Adverse events associated with clinical studies
Alsawady M, Borecka O & Llewellyn S (ISPOR US, 2023)
Patient feedback on the use of reward programs
Alsawady M, Amini F, Vincent S, Llewellyn S (ISPOR US, 2023)
Effectiveness of prostate cancer screening
Llewellyn S (ISPOR US, 2023)
Engagement statistics in real-world studies
Day L, Lau J, Quinn C (ISPOR EU, 2022)
Adherence rates in real-world studies
Borecka O, Ofori A & Llewellyn S (ISPOR EU, 2022)
Digital diagnosis validation feature
Vincent S, Borecka O, Llewellyn S & Bottomley C (ISPOR EU, 2022)
White paper: Patient-centric real-world evidence
The power of technology in real-world evidence generation
In today’s healthcare market, patient-centric evidence is crucial for product differentiation and success.
Diagnosis validation in patient-centered research
Llewellyn S, Bottomley C, Borecka O (National PROMs Annual Research Conference, 2022)
Patient-centricity with digital real-world evidence
When it comes to drug discovery there is a wealth of insight to be gained from the patient community. Involving patients in the design and development of real-world evidence studies, which in turn complement traditional clinical trials by demonstrating patients’ lived experience of the condition and its treatments, can be a powerful means of emphasizing patient-centricity in drug development. In this article, the authors explore the technicalities of engaging patients in real-world evidence studies.
Building a bridge between patient and pharma
To what extent does the scientific relevance of a healthcare research question parallel its relevance from the patient’s perspective? In this article, we use a live example of a real-world study investigating the experiences of individuals living with Charcot-Marie-Tooth disease (CMT) to demonstrate the wide-reaching value of patient involvement in study design and development.
Reimbursement of digital therapeutics
A comparison of the reimbursement of digital therapeutics in France, Germany, and the UK. Presented at ISPOR EU 2021.
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (Global Genes RARE Patient Advocacy Summit, 2022)
Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)
This manuscript was published in BMJ Open in May 2023. It discusses the patient-reported impact of myasthenia gravis in the real world using findings from a digital observational survey-based study (MyRealWorld MG).See here for more of our publications.Author Author...
Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG
Dewilde S, Philips G, Paci S, Beauchamp J, Chiroli S, Quinn C, Day L, Larkin M, Palace J, Berrih-Aknin S, Claeys K, Muppidi S, Mantegazza R, Saccà F, Meisel A, Bassez G, Murai H, Janssen MF (BMJ Open, 2022)
WHITE PAPER: Unleashing the potential of real-world evidence in rare diseases
Murthy A, Mantegazza R, Larkin M, Friconneau M, Annemans L, Van Damme B, Sokol T, Emond S (2022)
Digital data collection to measure the impact of myasthenia gravis on patients’ utility values in the real world
Dewilde S, Kousoulakou H, Janssen M, Claeys K, Friconneau M, Jacob S, Meisel A, Day L, Quinn C, Larkin M, Leighton T, Phillips G, Paci S (Value in Health, 25(1): S246)
Real-world patient-reported impact of myasthenia gravis: initial data from the MyRealWorld™ MG study
This poster was presented in October 2021 at the AANEM annual meeting. It shares the initial data from the MyRealWorld™ MG study about the impact of myasthenia gravis on the daily lives of patients.See here for more of our publications on myasthenia gravis and other...
Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
Berrih-Aknin S, Claeys K, Law N, Mantegazza R, Murai H, Sacca F, Dewilde S, Janssen M, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (BMJ Open 2021;11:e048198. doi: 10.1136/bmjopen-2020-048198)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Assouly P, Berrih-Aknin S, Claeys K, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J (Muscle & Nerve, 62: S65-S66)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Palace J, Assouly P, Claeys KG, Saccà F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (ABN, 2020)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Meisel A, Assouly P, Berrih-Aknin S, Claeys KG, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (DGN, 2020)
What role does caregiver burden play in health technology assessment bodies’ appraisals of highly specialized technologies?
Ofori A, Llewellyn S, Bottomley C, Kousoulakou H (ISPOR EU, 2020)
Sickle cell disease patient registries are not collecting enough data
Llewellyn S, Doe A, Åkesson C, Kousoulakou H, Quinn C, Larkin M (ISPOR US, 2020)
Complexities in localizing participant profile survey questions in real-world studies for smartphone apps
Kretz S, Richards A (Quality of Life Research, 29: S135)
The clinical and cost-effectiveness of supplemental parenteral nutrition in oncology
Webb N, Fricke J, Hancock E, Trueman D, Ghosh S, Winstone J, Miners A, Shepelev J, Valle JW (ESMO Open. 2020 Jun 1;5(3):e000709)
Challenges with using paper format legacy Patient Reported Outcome Measures (PROMs) in digital real-world evidence (RWE) studies for apps
Kretz S, Spinage C, Bottomley C (Quality of Life Research, 29: S137)
Innovative partnerships with patients and patient advocacy groups in digital observational rare disease studies.
Llewellyn S,Bagshaw E,Williams H,Larkin M (RARE Patient Advocacy Summit; 2019 Sep 18-20; San Diego, CA, US)
Melanoma
What is the consistency of response to rimegepant at a group level in the acute treatment of migraine in UK adult patients? Protocol for a real-world, patient-centred study
O’Neil G, Abraham L, Pawinski R, Nakajima K, Bagshaw E, Fellows A, Llewellyn S, Lambru G (EHC, 2025)
Assessment of stable chronic bronchitis improvement with adjunctive long-term Mucinex® use via the cough and sputum assessment questionnaire (CASA-Q)
Divel C, Borecka O, Llewellyn S, Spangenthal S (CHEST, 2025)
Adjunctive long-term use of Mucinex® leading to improvement in stable chronic bronchitis and patient’s quality of life: A case report.
Spangenthal S, Divel C, Borecka O, Llewellyn S (Medical Reports, 2025)
Real-world use of Vitaccess Real™ platform to assess quality of life impact with long-term use of Mucinex® in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Llewellyn S (ISOQOL, 2025)
Multiple myeloma caregiver costs and disabilities data for economic modelling and HTA submissions
Alsawady M, Tarnowska R, Kudlac A, Vincent S, Melrose D, Lied-Lied A (ISPOR Europe, 2025)
A targeted review of caregiver experiences of CAR-T therapy in ambulatory settings
Ringland C, Bagshaw E, Llewellyn S, Pugh G (ISPOR Europe, 2025)
Extrapulmonary disease burden and impact of cystic fibrosis (CF) on productivity in people with CF (pwCF) aged >12 years not treated with CF transmembrane conductance regulator modulators (CFTRm): interim analysis (IA) of the HUBBLE study
Elborn S, Mainz J, Abbott J, Carr S, Sole A, Costa S, Ganapathy V, Arteaga-Solis E, Liu J, Yuan J, Thorat T, Llewellyn S, Larkin M, De Iorio F, Kaplowitz H (ITS Annual Scientific Meeting, 2021)
Patient‑ and Caregiver‑Reported Burden of Transfusion‑Dependent β‑Thalassemia Measured Using a Digital Application
Paramore C, Levine L, Bagshaw E, Ouyang C, Kudlac A, Larkin M (Springer Nature’s, The Patient – Patient-Centered Outcomes Research, 2020)
Patient-reported burden of transfusion-dependent Beta-Thalassemia in the USA and UK measured using a digital app
Paramore C, Helfer J, Levine L, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Deprivation and access to care among patients with melanoma in the UK
Quinn C, Larkin M, Jacobs I, Kandola S, Smoyer K (ISPOR EU, 2022)
Using patient-reported data to estimate costs associated with melanoma
Javed M, Quinn C, Amini F, Boxell E (National PROMs Annual Research Conference, 2022)
Symptoms and side effects associated with melanoma treatment
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL 2021)
Contextualizing qualitative analysis with quantitative data on the symptoms and side effects associated with melanoma treatment in the real world – the value of patient registries
Ofori A, Ravindra H, Hall K, Bottomley C, Quinn C, Larkin M, Heinrich M (ISOQOL, 2021)
How do patients manage side effects during melanoma treatment? The importance of social support and doctor–patient communication
Ofori A, Hall K, Bottomley C, Larkin M, Heinrich M (The Christie School of Oncology, Melanoma Study Day, 2021)
Rewarding participants through charitable donations to Melanoma UK motivates recruitment and engagement: a bring your own device study example
Hall K, Ouyang C, Chilongo A, Llewellyn S, Quinn C, Larkin M (Quality of Life Research; 29: S91-S92)
Patient-centricity in action: using patient feedback to adapt the PRO-CTCAE instrument in a real-world study
Llewellyn S, Hall K, Camidge L, Bottomley C, Nixon A, Larkin M (Quality of Life Research, 29: S91)
Melanoma patient registries: a targeted review and comparison of datasets
Hall K, Llewellyn S, Bottomley C, Larkin M (Quality of Life Research, 29: S113)
Assessing the value of real-world evidence in melanoma in health technology assessment appraisals
Llewellyn S, Doe A, Quinn C (DIA Europe, 2020)
Targeted review of adverse events associated with treatments for stage 3 and 4 melanoma
Llewellyn S, Bagshaw E, Åkesson C, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Adverse events associated with immunotherapies used in the treatment of stage 3 and 4 melanoma
Llewellyn S, Åkesson C, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for melanoma in the UK measured using a digital ‘bring your own device’ platform
Larkin J, Cannon D, Nuttall G, Au L, Hunter N, Spain L, Turajlic S, Åkesson C, Llewellyn S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2019)
Real-world HRQL in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (PROMS Conference, 2018)
Taking the burden out of symptom reporting in patients with melanoma using a digital real-world evidence platform
Au L, Nuttall G, Cannon D, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Larkin J, Wiseman T (ISPOR US, 2018)
Real-world EQ-5D-5L utility values in patients with melanoma derived using a digital ‘Bring-Your-Own-Device’ platform
Larkin J, Nuttall G, Cannon D, Au L, Spain L, Hunter N, Turajlic S, Nixon A, Kousoulakou H, Larkin M, Wiseman T (ISPOR US, 2018)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-reported severity of pain interference in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (ANNEM 2023)
Patient-reported symptom severity of CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Lower extremity + upper limb disability in CMT1A
Thomas FP, Attarian S, Sevilla T, Genovese F, Gray AJ, Bull S, Tanesse D, Moore A, Hollett C, Paoli X, Day L, Kudlac A, Lau JKL, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2023)
Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A
Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray AJ, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Sénéchal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 2022 24(1): 7–17 )
Depression in patients with Charcot-Marie-Tooth
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Patient-reported symptom burden of Charcot-Marie-Tooth_PNS_2022
Thomas FP, Attarian S, Mascaró RS, Genovese F, Gray A, Bull S, Tanesse D, Hollett C, Moore A, Boutalbi Y, Paoli X, Day L, Llewellyn S, Ouyang C, Larkin M (2022)
Work impacts in patients with Charcot-Marie-Tooth_PNS_2022
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Disease burden and quality of life in patients with Charcot-Marie-Tooth disease type 1A, 2, 4 and X in Europe and the US measured using a digital app
Ziemssen T, Thomas FP, Sivera R, Saporta M, Genovese F, Gray A, Hollett C, Moore A, Rego M, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Larkin M, Ouyang C, Quinn C (Muscle & Nerve, 62: S69-S70)
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study
Thomas FP, Saporta M, Attarian S, Sevilla T, Mascaró RS, Fabrizi GM, Genovese F, Gray A, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Monteiro K, Paoli X, Llewellyn S, Larkin M, Boutalbi Y (Neurodegenerative Disease Management, 2021; 11(1): 21-33)
Patient-reported symptom burden of Charcot-Marie-Tooth disease Type 1a in Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343)
Patient-reported burden of Charcot-Marie-Tooth disease Type 1a In Europe and the US measured using a digital app
Thomas FP, Attarian S, Gray A, Hollett C, Moore A, Boutalbi Y, Monteiro K, Paoli X, Hall K, Llewellyn S, Ouyang C, Larkin M (Value in Health, 23(1): S343-344)
Productivity losses in people with Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world evidence study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Healthcare resource use in Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Demographics and treatment patterns for Charcot-Marie-Tooth disease in the EU and US: interim results from an international digital real-world study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Bagshaw E, Kousoulakou H, Larkin M (ISPOR EU, 2019)
Diversity in the Charcot–Marie–Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Thomas FP, Attarian S, Sevilla T, Gray A, Hollett C, Moore A, Bertrand V, Boutalbi Y, Larrazet M, Monteiro K, Paoli X, Llewellyn S, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment of the Charcot-Marie-Tooth disease population in the United Kingdom and United States: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Monteiro K, Bagshaw E, Kousoulakou H, Larkin M (AANEM, 2019)
Treatment patterns for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Care standards for Charcot-Marie-Tooth disease in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Diversity in the Charcot-Marie-Tooth disease population in the UK and US: insights from a digital real-world observational study
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (EAN, 2019)
Digitally assessed real-world diversity in people with Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Digitally assessed patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (PNS, 2019)
Patient-reported real-world care standards for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Patient-reported real-world treatment patterns for Charcot-Marie-Tooth disease in the UK and US assessed using a digital ‘bring your own device’ platform
Ziemssen T, Attarian S, Thomas FP, Moore A, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Bagshaw E, Kousoulakou H, Larkin M (ISPOR US, 2019)
Development of a digital app and innovative recruitment for an international real-world observational study in Charcot-Marie-Tooth disease
Ziemssen T, Attarian S, Thomas FP, Tanesse D, Paoli X, Bertrand V, Boutalbi Y, Kousoulakou H, Larkin M (ISPOR EU, 2018)
Patient-reported impact of adult-onset ALSP
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Caregiving burden among caregivers of people with myasthenia gravis
Dewilde S, Tollenaar NH, Boulanger P, Archer A, Pardo R, Cortés-Vicente E, Mantegazza R, Vanoli F, Lehnerer S, Pawlitzki M, Heinrich M, De Ruyck F, Phillips G, Paci S (Orphanet Journal of Rare Diseases, 2025)
Harnessing digital measures of physical function: findings from a targeted literature review
Aggio D, Ringland C, Amini F, Bowmar E, Llewellyn S (ISPOR, 2025)
A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry
Amini F, Lawrence J, Vincent S, Penduka T, Hall G, Fellows A, Larkin M, Scowcroft A, Beau Lejdstrom R, Savic N, Jacob S, Habib A, Saccà F, Hayes A (AAN, 2025 and MGFA International, 2025)
The interplay between CASA-Q domains: insights from a real-world study investigating the effects of extended-release guaifenesin in stable chronic bronchitis
Spangenthal S, Divel C, Borecka O, Fellows A, Llewellyn S (ACC, 2025)
Adjunctive long-term Mucinex® use leading to improvement in stable chronic bronchitis and decreased health care resource utilization – a case report
Divel C, Borecka O, Llewellyn S, Patel P, Shea T, Adeleke M, Spangenthal S (ATS, 2025)
Modified Delphi panel on advanced ALK-positive NSCLC patient profiling, treatment landscape, and clinical guidelines in the UK
Patel A, Tsang C, Baig LE, Llewellyn S, Baijal S (BTOG, 2025)
Caregiver-reported quality of life of patients with MCT8 deficiency: Results from a cross-sectional survey
Ofori A, Larkin M, Georges N (ISPOR Europe, 2024)
Quality of Life Impact of Caregiving for Patients with MCT8 Deficiency: Results from a Cross-Sectional Survey
Ofori A, Larkin M, Georges N (ISPOR EU, 2024)
Quality of patient engagement activities in health economics and outcomes research: insights from the ISPOR community
Llewellyn S, Qiao N, Masurkar P, Botto-van Bemden A, Roydhouse J, Perfetto E (Value & Outcomes Spotlight, 10(6):41-45)
Development of two conceptual models to describe the patient and caregiver reported impacts of living with X-linked myotubular myopathy (XLMTM)
Borecka O, Lawrence J, Llewellyn S (ISOQOL, 2024)
Leveraging SMS for patient-reported data collection
Llewellyn S, Amini F (ISOQOL, 2024)
Duchenne muscular dystrophy (DMD) patient vignettes development methodology
Merla V, Posner N, Borecka O, Vincent S (EAN, 2024)
Real world effectiveness of guaifenesin ER in tackling mucus hypersecretion in stable chronic bronchitis
Spangenthal S, Llewellyn S, Borecka O, Pollack C, Adeleke M, Kulasekaran A, Birring S, Mazzone S, Shea T (The Thirteenth London International Cough Symposium, 2024)
Patient-reported outcome measures used in interventional studies of irritable bowel syndrome: a targeted review
Lawrence J, Boxell E (National PROMS UK, 2024)
Outcomes and interventions in clinical studies investigating pharmacological therapies for the treatment of transthyretin amyloidosis: a targeted review
Alsawady M, Borecka O (National PROMS UK, 2024)
Diagnosis Validation in Patient-Centered Research
Alsawady M, Lawrence J, Amini F, Vincent S (Value and Outcomes Spotlight, 2024)
Assessing adherence in real-world studies
Ofori A, Octavia Borecka O, Llewellyn S
Approaches and challenges in developing caregiver vignettes in rare, progressive, and fatal childhood conditions: learnings from vignette development in Duchenne muscular dystrophy (DMD)
Llewellyn S, Borecka O, Bottomley C, Richardson L, Biggane A (ISOQOL, 2023)
Caregiver burden in growth hormone deficiency: a targeted review
Amini F, Alsawady M, Borecka O, Bottomley C
(ISPOR EU, 2023)
“I’ve got a text!” Feedback from prospective participants on the use of SMS surveys in real-world studies
Lawrence J, Amini F, Monera-Penduka TG, Llewellyn S
(ISPOR EU, 2023)
Vitamin D as an adjunct therapy in the treatment of atopic dermatitis: a targeted review
Borecka O, Lawrence J, Llewellyn S (ISPOR EU, 2023)
A targeted review of pharmacological therapies for the treatment of chronic spontaneous urticaria
Lawrence J, Borecka O, Alsawady M, Sauca M, Kallmes K, Llewellyn S (ISPOR EU, 2023)
Adverse events associated with clinical studies
Alsawady M, Borecka O & Llewellyn S (ISPOR US, 2023)
Patient feedback on the use of reward programs
Alsawady M, Amini F, Vincent S, Llewellyn S (ISPOR US, 2023)
Effectiveness of prostate cancer screening
Llewellyn S (ISPOR US, 2023)
Engagement statistics in real-world studies
Day L, Lau J, Quinn C (ISPOR EU, 2022)
Adherence rates in real-world studies
Borecka O, Ofori A & Llewellyn S (ISPOR EU, 2022)
Digital diagnosis validation feature
Vincent S, Borecka O, Llewellyn S & Bottomley C (ISPOR EU, 2022)
White paper: Patient-centric real-world evidence
The power of technology in real-world evidence generation
In today’s healthcare market, patient-centric evidence is crucial for product differentiation and success.
Diagnosis validation in patient-centered research
Llewellyn S, Bottomley C, Borecka O (National PROMs Annual Research Conference, 2022)
Patient-centricity with digital real-world evidence
When it comes to drug discovery there is a wealth of insight to be gained from the patient community. Involving patients in the design and development of real-world evidence studies, which in turn complement traditional clinical trials by demonstrating patients’ lived experience of the condition and its treatments, can be a powerful means of emphasizing patient-centricity in drug development. In this article, the authors explore the technicalities of engaging patients in real-world evidence studies.
Building a bridge between patient and pharma
To what extent does the scientific relevance of a healthcare research question parallel its relevance from the patient’s perspective? In this article, we use a live example of a real-world study investigating the experiences of individuals living with Charcot-Marie-Tooth disease (CMT) to demonstrate the wide-reaching value of patient involvement in study design and development.
Reimbursement of digital therapeutics
A comparison of the reimbursement of digital therapeutics in France, Germany, and the UK. Presented at ISPOR EU 2021.
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (ISPOR EU, 2022)
Protocol for the first global study capturing the patient-reported impact of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in the real-world setting using a smartphone application
Pontius A, Meier A, Papapetropoulos S, Lynch DS, Edwards H, Day L, Llewellyn S, Quinn C (Global Genes RARE Patient Advocacy Summit, 2022)
Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)
This manuscript was published in BMJ Open in May 2023. It discusses the patient-reported impact of myasthenia gravis in the real world using findings from a digital observational survey-based study (MyRealWorld MG).See here for more of our publications.Author Author...
Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG
Dewilde S, Philips G, Paci S, Beauchamp J, Chiroli S, Quinn C, Day L, Larkin M, Palace J, Berrih-Aknin S, Claeys K, Muppidi S, Mantegazza R, Saccà F, Meisel A, Bassez G, Murai H, Janssen MF (BMJ Open, 2022)
WHITE PAPER: Unleashing the potential of real-world evidence in rare diseases
Murthy A, Mantegazza R, Larkin M, Friconneau M, Annemans L, Van Damme B, Sokol T, Emond S (2022)
Digital data collection to measure the impact of myasthenia gravis on patients’ utility values in the real world
Dewilde S, Kousoulakou H, Janssen M, Claeys K, Friconneau M, Jacob S, Meisel A, Day L, Quinn C, Larkin M, Leighton T, Phillips G, Paci S (Value in Health, 25(1): S246)
Real-world patient-reported impact of myasthenia gravis: initial data from the MyRealWorld™ MG study
This poster was presented in October 2021 at the AANEM annual meeting. It shares the initial data from the MyRealWorld™ MG study about the impact of myasthenia gravis on the daily lives of patients.See here for more of our publications on myasthenia gravis and other...
Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
Berrih-Aknin S, Claeys K, Law N, Mantegazza R, Murai H, Sacca F, Dewilde S, Janssen M, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (BMJ Open 2021;11:e048198. doi: 10.1136/bmjopen-2020-048198)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Assouly P, Berrih-Aknin S, Claeys K, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J (Muscle & Nerve, 62: S65-S66)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Palace J, Assouly P, Claeys KG, Saccà F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (ABN, 2020)
Capturing the patient-reported impact of myasthenia gravis in the real-world setting using a smartphone application
Meisel A, Assouly P, Berrih-Aknin S, Claeys KG, Murai H, Palace J, Saccà F, Baggi F, Bagshaw E, Kousoulakou H, Larkin M, Beauchamp J, Leighton T, Paci S (DGN, 2020)
What role does caregiver burden play in health technology assessment bodies’ appraisals of highly specialized technologies?
Ofori A, Llewellyn S, Bottomley C, Kousoulakou H (ISPOR EU, 2020)
Sickle cell disease patient registries are not collecting enough data
Llewellyn S, Doe A, Åkesson C, Kousoulakou H, Quinn C, Larkin M (ISPOR US, 2020)
Complexities in localizing participant profile survey questions in real-world studies for smartphone apps
Kretz S, Richards A (Quality of Life Research, 29: S135)
The clinical and cost-effectiveness of supplemental parenteral nutrition in oncology
Webb N, Fricke J, Hancock E, Trueman D, Ghosh S, Winstone J, Miners A, Shepelev J, Valle JW (ESMO Open. 2020 Jun 1;5(3):e000709)
Challenges with using paper format legacy Patient Reported Outcome Measures (PROMs) in digital real-world evidence (RWE) studies for apps
Kretz S, Spinage C, Bottomley C (Quality of Life Research, 29: S137)
Innovative partnerships with patients and patient advocacy groups in digital observational rare disease studies.
Llewellyn S,Bagshaw E,Williams H,Larkin M (RARE Patient Advocacy Summit; 2019 Sep 18-20; San Diego, CA, US)
Vitaccess Publications
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