“Watching time tick by…”: Decision making for Duchenne muscular dystrophy trials

Abstract

Objective This interview study explored clinicians’ perspectives and parents’ decision making about children’s participation in Duchenne muscular dystrophy (DMD) clinical trials. Methods Data from semi-structured interviews conducted with clinicians and parents in U.S. or Canada were assessed using thematic analysis. Results Eleven clinicians involved in ten trials and fifteen parents involved in six trials were interviewed. Parents described benefit-risk assessments using information from advocacy, peers, professionals, and sponsors. Strong influence was attributed to the progressive nature of DMD. Most expected direct benefit. Few considered the possibility of trial failure. Most made decisions to participate before the informed consent (IC) process, but none-the-less perceived informed choice with little to lose for potential gain. Clinicians described more influence on parental decisions than attributed by parents. Clinicians felt responsible to facilitate IC while maintaining hope. Both clinicians and parents reported criticisms about the IC process and regulatory barriers. Conclusions The majority of parents described undertaking benefit-risk assessments that led to informed choices that offered psychological and potential disease benefits. Parents’ high expectations influenced their decisions while also reflecting optimism. Clinicians felt challenged in balancing parents’ expectations and likely outcomes. Prognosis-related pressures coupled with decision making prior to IC suggest an obligation to ensure educational materials are understandable and accurate, and to consider an expanded notion of IC timeframes. Anticipatory guidance about potential trial failure might facilitate parents’ deliberations while aiding clinicians in moderating overly-optimistic motivations. Regulators and industry should appreciate special challenges in progressive disorders, where doing nothing was equated with doing harm.

Fragile X syndrome clinical trials: exploring parental decision‐making

“…We found that parents of children with FXS may be more risk averse to side effects compared with what has been observed in parents of children with lifethreatening conditions (Cartwright et al 2011;Deatrick et al 2002;Peay et al 2016). Parents of children with FXS, a chronic and stable condition, may be particularly resistant to risking their child's physical health.…”

“…Parents of children with life‐threatening conditions, conversely, are faced with relatively little to lose in this domain, and medication side effects may be perceived as less harmful for their child when compared with the untreated disease course (Peay et al . ).…”

“…; Peay et al . , ). We found that parents evaluated the potential benefit of the drug through their perception of the drug's mechanism of action and the degree of match between the trial outcome measures and their child's symptomatology.…”

“…Parents of children with FXS, a chronic and stable condition, may be particularly resistant to risking their child's physical health. Parents of children with lifethreatening conditions, conversely, are faced with relatively little to lose in this domain, and medication side effects may be perceived as less harmful for their child when compared with the untreated disease course (Peay et al 2016).…”

Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation

“…Health professionals who recruit should communicate the balance of benefits and risks associated with trial treatment, stressing negative physiological outcomes such as pain and fatigue. 272 Professionals should use questions and feedback to ensure that the child understands that they are participating in research, and that participation is voluntary; assent should be continuously renegotiated throughout the trial. 273 ii.…”