LoRusso Samantha, Eichinger Katy, Higgs Kiley, Lewis Leann, Walker Michaela, Albert James, Langer Michele, Tawil Rabi, Statland Jeffrey M, Kimminau Kim S
Department of Neurology (SL), Ohio State University Medical Center, Columbus, Ohio; Department of Neurology (KE. LL, RT), Strong Memorial Hospital, University of Rochester, Rochester, New York; Department of Neurology (KH, MW, JMS), University of Kansas Medical Center, Kansas City, Kansas; University of Missouri School of Medicine (KSK), Columbia, Missouri.
Neurol Clin Pract. 2021 Oct;11(5):e722-e726. doi: 10.1212/CPJ.0000000000001074.
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.
我们阐述了为克服面肩肱型肌营养不良症（FSHD）患者参与研究的障碍所做的努力，并提供了一份可在其他罕见神经系统疾病中复制的路线图。我们在“解决FSHD药物开发障碍临床试验准备阶段”（ReSolve）期间实施了一项参与计划，这是一项针对FSHD患者的为期18个月的多中心观察性研究。我们参与计划的要素包括在方案制定过程中开展焦点小组讨论、让患者参与ReSolve外部咨询委员会、成立患者咨询委员会以及与患者倡导组织合作。患者反馈促使研究方案进行了调整，并改变了招募和保留方法。患者参与确保了患者的声音能为试验设计和实施的多个方面做出贡献。我们的参与工作例证了在FSHD中如何与患者及家属实现合作，并且由此产生的路线图流程可能在其他罕见神经系统疾病中得以复制。
