Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders

Abstract

We found high parental interest in pediatric neuromuscular trials that was tempered by concerns about the potential for randomization to a placebo arm. Participants perceived that their trial participation would be facilitated by additional education and guidance from their clinicians. Yet, intentions were negatively associated with frequency of provider communication, perhaps reflecting waning parental interest with a greater understanding of limitations in trial access, increased sophistication in their understanding of trial design, and appreciation of potential burden. To support parents' informed decisions, it is important to educate them to evaluate the quality of research, as well as providing lay information explaining the use of placebo, trial processes, and potential barriers to long-term drug access. Our findings should inform the development of targeted educational content, clinician training, and decision support tools.

Profiling trial burden and patients’ attitudes to improve clinical research in epidermolysis bullosa

“…(Fig. S 5 ) It is also contrary to previous data identifying physicians’ recommendation to play a key role in patients’ beliefs about clinical trials and in their decision [ 34 , 40 – 43 ]. Former studies likewise highlighted the relevance of social media.…”

Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents

“…These findings, which are the first focused on gene therapy for Duchenne, expand upon prior studies of clinical trial preferences and attitudes. Prior studies found similar high interest and intentions to participate in non-gene-therapy trials, general optimism regarding scientific advancement juxtaposed with worry about disease progression, and concerns about access to new therapeutics under trial [33–36]. While two prior studies assessed tolerance for serious but non-fatal adverse trial events [37, 38], this is the first study to explore tolerance for risk of therapy-related death.…”

236th ENMC International Workshop Bone protective therapy in Duchenne muscular dystrophy: Determining the feasibility and standards of clinical trials Hoofddorp, The Netherlands, 1–3 June 2018

“…However, the use of placebo, insufficient information on risks of novel therapies, and inadequate information about study requirements were identified as major barriers for the participation in clinical trials. Confidence that the research will improve understanding of bone health and osteoporosis, and guarantee that treatment will be provided after a successful trial were deemed as facilitators [24] .…”