G.P.302: Perceived barriers and facilitators of participation in clinical trials for Duchenne and Becker muscular dystrophy

Abstract

Introduction: Industry, clinicians, advocacy, and families have a stake in the success of clinical trials (CTs). Understanding decision-makers' perceptions of CTs inform efforts to improve recruitment; this is particularly relevant for rare diseases like Duchenne/Becker muscular dystrophy (DBMD). Methods: The study team employed a community-based participatory research approach. US/Canadian parents of children with DBMD who never participated in a CT were recruited for an online survey through Parent Project Muscular Dystrophy and clinics. Participants answered questions about CT preferences and perceived barriers and facilitators. Results: Among the first 99 participants with children age 4–12, 65% wanted their child in a CT, 32% were unsure, and 3% did not. The most endorsed barriers were: possibility of receiving placebo (46% very or moderately true); insufficient information about risks (27%); the possibility of an unsuccessful trial (25%); child may not benefit (25%); and insufficient information about day-to-day requirements (25%). Parents unsure about CT participation endorsed barriers at notably different frequencies than those wanting participation for: insufficient risk information (55% vs. 13%) and child not benefiting (39% vs. 16%). Highly-endorsed facilitators of increased future CT interest were: guaranteed treatment access after CT (82%); having confidence CT would improve DBMD understanding (76%); agreeing with CT goals (75%); treating doctor's suggestion (72%); perceiving a good researcher reputation (71%). Conclusions: Many facilitators were highly rated for increasing interest in CT participation, supporting adaptive trial design and education and outreach. Parents unsure about participation may have more needs for CT information. The most-endorsed facilitator was treatment access after CT, which is very difficult to achieve. Sponsors should increase outreach through community forums and clinicians to clarify CT processes, personnel, and goals.