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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.

Building a bridge between patient and pharma: the CMT story

Amini F (Rare Revolution Magazine, 2022)

See here for more of our publications on Charcot-Marie-Tooth disease and other subjects.

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Read about Fatemeh, our Associate Medical Writer, on our Who we a re page.

Fatemeh Amini

September 8, 2025
Publications

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Supporting evidence-generation with expertise

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A Novel International Patient Registry in Chronic Inflammatory Demyelinating Polyneuropathy Linking Clinical and Patient-Reported Outcomes Data: The Vitaccess Real CIDP (VRCIDP) Registry

Bowmar E, Larkin M, Habib A, Di Salvo N, Rajabally Y (AAN 2026)

Impact of Caring for Patients with Monocarboxylate Transporter 8 (MCT8) Deficiency on Caregivers’ Sleep: Results from an International Cross-Sectional Study

Ofori A, Larkin MJ, Georges N (PES 2025)

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)

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Advanced patient-centric, science-driven research

Observational Studies

Qualitative Research

Registries

Health Utilities & Preference Studies

Quantitative Research

Analytics & Data Visualization

Patient-reported Outcomes

Translation & Linguistic Validation

Publication Support

Patient & Caregiver Burden Studies

Publications

Blogs

Case studies

White papers

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Study datasets

Building a bridge between patient and pharma

Building a bridge between patient and pharma: the CMT story

Supporting evidence-generation with expertise

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