publications
Working in collaboration with our sponsors, clinical experts, and patient groups, we distill the data from our studies into compelling publications in peer-reviewed journals and conference posters.
Take a look at our publications archive to see how our work amplifies the voices of our study participants.
Alopecia areata: targeted review of adverse events associated with clinical studies of treatments
Alsawady M, Borecka O, Llewellyn S (ISPOR US, 2023)
“Cash or credit?” Feedback from patients on the use of reward programs in digital research
Alsawady M, Amini F, Vincent S, Llewellyn S (ISPOR US, 2023)
Effectiveness of prostate cancer screening in reducing adult mortality: a quasi-systematic review
Llewellyn S (ISPOR US, 2023)
The Past, Present, and Future of Sickle-cell Disease Registries: Findings from a Global Review
Llewellyn S, Amini F, Borecka O, Ofori A (Pharma Focus Asia, 2023)
MANUSCRIPT: Sickle cell disease: a global patient registry review
Borecka O, Ofori A, Amini F, Llewellyn S (Future Rare Diseases, 2022)
Challenges in presenting engagement statistics in real-world digital studies
Day L, Lau J, Quinn C (ISPOR EU, 2022)
“Please complete your surveys”: a targeted review of adherence rates in real-world studies using digital technologies
Borecka O, Ofori A, Llewellyn S (ISPOR EU, 2022)
“How easily could you obtain proof of diagnosis for your medical condition?” Feedback from individuals with chronic medical conditions on a digital diagnosis validation feature
Vincent S, Borecka O, Llewellyn S, Bottomley C (ISPOR EU, 2022)
WHITE PAPER: Patient-centric real-world evidence: benefits, uses, and the power of digital technology
Amini F, Bagshaw E, Bottomley C, Larkin M (2022)
When is a patient a patient? Diagnosis validation in patient-centered research
Llewellyn S, Bottomley C, Borecka O (National PROMS UK, 2022)
Tips of the Trade: Delivering Patient-Centricity With Digital Real-World Evidence
Building a bridge between patient and pharma: the CMT story
A comparison of the reimbursement of digital therapeutics in France, Germany, and the UK
Ofori A, Monnickendam G (ISPOR EU, 2021)
Is there an optimal way to estimate healthcare resource use?
eCOA migration for bring-your-own-device technology: comparison of user testing versus screenshot review
Richards A, Kretz S, Miller F (ISOQOL, 2021)
Comparative study of linguistic validation developer-prescribed processes involved in the development of patient-reported outcomes
Miller F, Kretz S, Richards A, Spinage C (ISOQOL, 2021)
Which data are migraine patient registries collecting? A targeted review
Ofori A, Llewellyn S, Bottomley C, Kousoulakou H (Value in Health, 23(2): S746)
Gamification – the future of real-world patient-reported studies? Analysis of the ethical and legal challenges of applying gamification in observational studies using smartphone apps: a review of the UK and France
Richards A, Bottomley C (Quality of Life Research, 29: S129)
Treatment patterns and healthcare resource use in primary hypercholesterolaemia and mixed dyslipidaemia: results of a UK Delphi panel
A conceptual framework for the approach to the evluation of oncology survival benefit in health technology assessment
Monnickendam G, Larkin M, Larkin J, Quinn C (ISPOR EU, 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)
MANUSCRIPT: 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
MANUSCRIPT: Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)
Berrih-Aknin S, Palace J, Meisel A, Claeys K, Muppidi S, Saccà F, Amini F, Larkin M, Quinn C, Beauchamp J, Philips G, De Ruyck F, Ramirez J, Paci S (BMJ Open 2023)
MANUSCRIPT: 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
Berrih-Aknin S, Claeys KG, Friconneau M, Mantegazza R, Meisel A, Murai H, Palace J, Saccà F, Bagshaw E, Larkin M, Beauchamp J, De Ruyck F, Paci S, Phillips G (AANEM, 2021)
MANUSCRIPT: 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)
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)
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 2022 RARE Patient Advocacy Summit)
Cystic fibrosis
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)
Beta-thalassemia
MANUSCRIPT: 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)
Charcot-Marie-Tooth
MANUSCRIPT: Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A: Findings From an Observational Digital Lifestyle Study
Thomas FP, Saporta MA, Attarian S, Sevilla T, Sivera R, Fabrizi GM, Genovese F, Gray A, Bull S, Tanesse D, Rego M, Moore A, Hollett C, Paoli X, Senechal T, Day L, Ouyang C, Llewellyn S, Larkin M, Boutalbi Y (Journal of Clinical Neuromuscular Disease, 24(1): 7-17 )
Depression in patients with Charcot-Marie-Tooth disease type 1A (CMT1A): findings from a real-world digital study
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Patient-reported symptom burden of Charcot-Marie-Tooth disease type 1A (CMT1A): findings from a real-world digital study
Thomas F, Sevilla T, Sivera R, Genovese F, Gray A, Paoli X, Sénéchal T, Day L, Llewellyn S, Larkin M, Boutalbi Y (PNS, 2022)
Work impacts in patients with Charcot-Marie-Tooth disease type 1A (CMT1A): findings from a real-world digital study
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)
MANUSCRIPT: 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 Europe, 2018)
Melanoma
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 in the UK: a digital registry
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 Europe, 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)
