Background: Patients with generalized myasthenia gravis (gMG) and care partners can bring valuable insights to guide research. However, use of patient and care partner perspectives to ensure a patient-centric approach in the real-world (RW) setting remains limited.
Objective: To advance patient-centricity in gMG research by obtaining patient and care partner input on a prospective, RW study for gMG.
Methods: Nine gMG patients and two care partners from the gMG Patient Engagement Research Council (PERC) participated in one of three virtual, semi-structured focus groups in March 2023. PERC represented a diverse group of patients with respect to time since diagnosis, disease severity, treatments, age, gender, and race/ethnicity. The 2-hour sessions focused on capturing the impressions, perceived challenges, and logistical burden around participating in a gMG RW study, as well as feedback on providing data via surveys and wearing monitoring devices.
Results: Based on the adjusted PERC insights-guided study design, both patients and care partners expressed interest to participate in research that advances medication development and treatment of gMG. Participants provided insight on logistics including travel, compensation, time commitment, and burden of data collection. The importance of “meeting patients where they are” in their disease journey to ensure completeness of patient-reported data was a key finding, such as including options to customize questionnaire reminders to suit different patient needs and lifestyles. Almost all (90.9%; 10/11) participants said they would consider joining a RW study to help both themselves and future generations.
Conclusions: Involving patients in the design of research is crucial in understanding how to ensure outcomes are patient-centric with a reasonable study burden. Insights from this innovative research design directly impacted the development of a large, global RW study, thus supporting recruitment goals, adherence to the study protocol, and the overall success and relevance of this research to patients with gMG.