Abstract
We describe our efforts to overcome barriers to patient engagement in facioscapulohumeral muscular dystrophy (FSHD), and offer a roadmap that can be replicated in other rare neurological disorders. We implemented an engagement plan during Clinical Trial Readiness to Solve Barriers to Drug Development for FSHD (ReSolve), an 18-month, multi-site, observational study of individuals with FSHD. Elements of our engagement plan included conducting focus groups during protocol development, patient involvement on the ReSolve external advisory committee, creation of a patient advisory committee, and collaboration with patient advocacy groups. Patient feedback led to adaptations in the study protocol and to changes in recruitment and retention methods. Patient engagement ensures the patient voice contributes to multiple aspects of trial design and implementation. Our engagement efforts exemplify how collaboration with patients and families can be accomplished in FSHD and the resultant roadmap process may be replicable in other rare neurological diseases.
- Received November 13, 2020.
- Accepted February 8, 2021.
- © 2021 American Academy of Neurology
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