P.2.15 Expectations and decision making in clinical trials for Duchenne and Becker muscular dystrophy

Abstract

Increased community knowledge of and access to clinical trials for Duchenne and Becker muscular dystrophy (DBMD) may amplify adaptive optimism, but also inappropriately high expectations. This study explored parental and clinician motivations and expectations for DBMD clinical trials. We interviewed 15 parents of children with DBMD and 11 clinicians participating in 10 clinical trials for DBMD. Interviews were transcribed and coded for thematic analysis. All parents hoped for, and most expected, direct benefit to their child; this was their primary motivator. Other motivators were a desire to affect the disease course; that “doing something is better than nothing;” and altruism. Most parents actively sought out a trial and described making their decision before the informed consent process (IC). The majority hoped for a cure but recognized this as unrealistic. Parents’ reported expectations were inconsistent in different contexts during the interviews. Some had difficulty differentiating between expectations and optimistic hopes. Some reported efforts to temper their expectations, or outside factors tempering expectations such as being in a placebo-controlled trial. Many parents described changes to expectations and hopes over time. Clinicians reported that families were motivated by desperation and hope for benefit. Altruism was a less important motivator. Clinicians’ motivations included optimism, enthusiasm for having more to offer, and personal commitment. Clinicians’ expectations ranged from modest gains in knowledge to clinical improvement. This study suggests the importance of interventions that help parents clarify expectations and identify inappropriately high expectations, and anticipatory guidance regarding challenges to optimistic hopes. As parents reported decision making before IC, interventions prior to IC may be most effective. Further studies are needed to identify predictors of inappropriately high expectations in DBMD clinical trials.