Design of and Early Insights From a Generalized Myasthenia Gravis Patient Engagement Research Council

ABSTRACT Background and Aims An exploratory focus group study was conducted to better understand the needs of patients living with generalized myasthenia gravis (gMG). Methods US‐based adults with gMG and caregivers of patients with gMG participated in a Patient Engagement Research Council between August 2022 and January 2023. The study consisted of a 15‐min prework survey, followed by virtual focus groups facilitated using a semi‐structured interview guide. Data concepts were identified using conversational analysis and by direct observation. All transcripts were coded based on concepts using a qualitative research analysis program (MaxQDA). Results 16 participants (13 patients, three caregivers) were recruited. Participants reported impact on daily activities, fatigue, and psychosocial problems. Many participants experienced delayed diagnosis and difficulty accessing specialist care. Participants described multiple barriers related to their gMG, including barriers to treatment, access‐related issues, and communication disconnect between patients and healthcare professionals. Achieving stable disease was the most important goal. There was a preference for the autonomy of self‐administered medications at home versus infusions. Study insights led to recommendations to guide patient and healthcare professional education. Conclusion The study illustrates the need to improve access to specialist care, achieve earlier diagnosis, prioritize patients' preferences in disease management, and develop treatments that improve outcomes without additional burden. Patient or Public Contribution The data collected in this study was provided by the focus group participants, which included patients and caregivers of those with myasthenia gravis.