RT Journal Article SR Electronic T1 A Roadmap to Patient Engagement JF Neurology: Clinical Practice FD Lippincott Williams & Wilkins SP e722 OP e726 DO 10.1212/CPJ.0000000000001074 VO 11 IS 5 A1 LoRusso, Samantha A1 Eichinger, Katy A1 Higgs, Kiley A1 Lewis, Leann A1 Walker, Michaela A1 Albert, James A1 Langer, Michele A1 Tawil, Rabi A1 Statland, Jeffrey M. A1 Kimminau, Kim S. A1 , YR 2021 UL http://cp.neurology.org/content/11/5/e722.abstract AB 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 neurologic disorders. We implemented an engagement plan during Clinical Trial Readiness to Solve Barriers to Drug Development for FSHD (ReSolve), an 18-month, multisite, 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 that 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 neurologic diseases.