"Watching time tick by…": Decision making for Duchenne muscular dystrophy trials.

Autor: Peay HL; Parent Project Muscular Dystrophy, Hackensack, NJ, USA; Department of Clinical Genetics, Leiden University Medical Centre, Leiden, The Netherlands. Electronic address: hpeay@rti.org., Scharff H; Parent Project Muscular Dystrophy, Hackensack, NJ, USA., Tibben A; Department of Clinical Genetics, Leiden University Medical Centre, Leiden, The Netherlands., Wilfond B; Treuman Katz Center for Pediatric Bioethics, Seattle Children's Research Institute, Seattle, WA, USA., Bowie J; Department of Health, Behavior & Society, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA., Johnson J; Parent Project Muscular Dystrophy, Hackensack, NJ, USA., Nagaraju K; Children's National Medical Center, Washington DC, USA., Escolar D; Kennedy Krieger Institute, Baltimore, MD, USA., Piacentino J; Parent Project Muscular Dystrophy, Hackensack, NJ, USA., Biesecker BB; Social and Behavioral Research Branch, National Human Genome Research Institute, Bethesda, MD, USA.
Jazyk: angličtina
Zdroj: Contemporary clinical trials [Contemp Clin Trials] 2016 Jan; Vol. 46, pp. 1-6. Date of Electronic Publication: 2015 Nov 09.
DOI: 10.1016/j.cct.2015.11.006
Abstrakt: 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.
(Copyright © 2015 Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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