Background: Generalized Myasthenia Gravis (gMG) is a rare, incurable, autoantibody mediated disorder with profound impact on those affected. Patients and care partners possess experiential knowledge that is unique and complementary to clinical development and real-world research which can improve research quality, relevance, and outcomes.
Objective: To describe the design and early insights from structured focus groups with the gMG Patient Engagement Research Council (PERC), including recruitment and participant demographics.
Methods: Recruitment of 12-14 adults residing in the United States with self-reported diagnosis of gMG and 2-4 care partners is occurring between August and December 2022. A MG-specific online screener, interview guide, and outreach materials were developed, and interviews were conducted to select participants. Variety in time since diagnosis, mix of disease severity/treatment experiences, early vs. later age at diagnosis, and a balance across age, gender and race/ethnicity is guiding recruitment. An initial virtual focus group will be conducted to introduce participants to the program and learn about disease journey, symptoms (fluctuations, most bothersome), diagnosis experience, daily functioning, quality of life, work/school impacts, outcomes of importance, and the burden of disease and treatments. This methodology will be replicated to explore additional topics through 2023.
Results: As of December, recruitment is almost complete with 12 participants with self-reported gMG and 2 care partners successfully recruited, with representation across race/ethnicity, gender, geographic residence, time since diagnosis, and treatment experience. The initial engagement is planned for January 2023 and insights will be reported.
Conclusions: Insights from the gMG PERC engagements will directly enhance research, help identify patient and provider educational needs, and guide communication strategies with implications for improving the quality of lives of those with gMG and their care partners.