title: "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.
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, NJ, USA; Department of Clinical Genetics, Leiden University Medical Centre, Leiden, The Netherlands. Electronic address: hpeay@rti.org.
authors.forename: Holly L
authors.initials: HL
authors.lastname: Peay
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, NJ, USA.
authors.forename: Hadar
authors.initials: H
authors.lastname: Scharff
authors.affiliation: Department of Clinical Genetics, Leiden University Medical Centre, Leiden, The Netherlands.
authors.forename: Aad
authors.initials: A
authors.lastname: Tibben
authors.affiliation: Treuman Katz Center for Pediatric Bioethics, Seattle Children's Research Institute, Seattle, WA, USA.
authors.forename: Benjamin
authors.initials: B
authors.lastname: Wilfond
authors.affiliation: Department of Health, Behavior & Society, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
authors.forename: Janice
authors.initials: J
authors.lastname: Bowie
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, NJ, USA.
authors.forename: Joanna
authors.initials: J
authors.lastname: Johnson
authors.affiliation: Children's National Medical Center, Washington DC, USA.
authors.forename: Kanneboyina
authors.initials: K
authors.lastname: Nagaraju
authors.affiliation: Kennedy Krieger Institute, Baltimore, MD, USA.
authors.forename: Diana
authors.initials: D
authors.lastname: Escolar
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, NJ, USA.
authors.forename: Jonathan
authors.initials: J
authors.lastname: Piacentino
authors.affiliation: Social and Behavioral Research Branch, National Human Genome Research Institute, Bethesda, MD, USA.
authors.forename: Barbara B
authors.initials: BB
authors.lastname: Biesecker
mesh_terms: D000293:Adolescent; D001291:Attitude of Health Personnel; D001294:Attitude to Health; D002170:Canada; D002648:Child; D002986:Clinical Trials as Topic; D003657:Decision Making; D005260:Female; D006801:Humans; D007258:Informed Consent; D008297:Male; D009042:Motivation; D020388:Muscular Dystrophy, Duchenne; D010290:Parents; D018579:Patient Selection; D019727:Proxy; D036301:Qualitative Research; D012108:Research Personnel; D012189:Retrospective Studies; D018570:Risk Assessment
keywords: Clinical trial; Consent; Duchenne muscular dystrophy; Proxy decision making