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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)

Recommendations for optimising the use, design and regulation of real-world evidence (RWE) collection based on an expert panel hosted by the Associazione Italiana Miastenia (AIM) on 1 March 2021.

See here for more of our publications on myasthenia gravis and other subjects.

Authors

Author profiles can be found on our Who we a re page.

Mark Larkin

September 3, 2025
Publications

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Supporting evidence-generation with expertise

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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)

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Study datasets

WHITE PAPER: Unleashing the potential of real-world evidence in rare diseases

Supporting evidence-generation with expertise

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