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.