Implementing a Hybrid, Real-World Study Design to Reduce Burden for Patients With Spinal Muscular Atrophy (SMA) Treated With Risdiplam


Clinical Management

Poster Number: S88


Truc Moyer, Genentech, Inc, Beverly Assman, MLS, Genentech, Inc

Approximately 1 in 10,000 individuals in the US are diagnosed with SMA – a rare, progressive neuromuscular disease that affects mobility, breathing, eating and swallowing. With the advent of disease-modifying therapies, including risdiplam (EVRYSDI®), patients with SMA are living longer, which makes understanding long-term treatment patterns and safety and effectiveness outcomes important. However, traditional study designs may present challenges due to patient dispersion and participation burden. The WeSMA study (NCT05232929), which assesses the long-term safety and effectiveness of risdiplam in pediatric and adult participants with all SMA types, seeks to develop a more inclusive, patient-centric and accessible clinical study for individuals with SMA.

To design a hybrid (in-person and virtual) study for all U.S.-based risdiplam-treated people living with SMA. The study aims to facilitate patient engagement, enrollment, retention and data collection.

All risdiplam-treated individuals with SMA that reside in the U.S. are eligible for the WeSMA study. Participants with access to a smartphone or tablet can self-enroll via a participant-facing electronic platform that enables a decentralized trial model. A virtual patient coordinating center provides support for participants using the electronic platform. Participants are asked to complete eConsent forms, attend telehealth visits, conduct remote and in-person assessments and complete study questionnaires/patient-reported outcomes via the electronic platform.

Of all participants enrolled in the WeSMA study, 75.8% enrolled in one of 25 active in-person sites in the first 23 months and 24.2% enrolled in the central virtual site in the first 8 months.

Hybrid study designs using patient-centric technology may expand accessibility and promote diverse participation while improving patient experience. This clinical study illustrates the impact modernized study designs may have on researchers’ ability to generate data from real-world settings that are more inclusive.