title: Developing a Patient-Centered Benefit-Risk Survey: A Community-Engaged Process.
abstract: OBJECTIVE
  To provide a community-engaged process to inform the design of a stated-preferences experiment. The process involved integrating patients and caregivers of people with Duchenne/Becker muscular dystrophy, advocates, clinicians, and the sponsor in conceptualizing and developing a benefit-risk survey on the basis of phase III trial results.
  
  
  METHODS
  Our community-engagement process for the development of a stated-preference survey included a set of five guiding principles with a foundation in the principles of community-engaged research. Engagement efforts were carried out through an informal network of three committees. Members of the leadership, stakeholder, and review committees comprised patients, caregivers, clinicians, advocacy leadership, and industry representatives.
  
  
  RESULTS
  Committee members participated in 15 hours of formal engagement including interviews and conference calls that ranged from 45 to 90 minutes, plus additional less-formal ad hoc communication. Committees comprised 20 individuals across three committees including adults with DMD (n = 6), parents of children with DMD (n = 6), clinicians (n = 3), members of research and advocacy organizations (n = 4), and an industry representative (n = 1). Community engagement informed attribute selection, survey length, word choice, and eligibility criteria. Challenges in the process included managing diverse stakeholder perspectives, time requirements, and the inherent tension between outcomes used in clinical trials versus attributes that correspond to patient- and family-relevant outcomes.
  
  
  CONCLUSIONS
  We demonstrated how community engagement can successfully influence study design to support the design of a relevant survey instrument that is ethical, acceptable, meaningful to the community, and enhances patient-centered benefit-risk assessment for regulatory decision making.
authors.affiliation: Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA. Electronic address: ilene.hollin@jhu.edu.
authors.forename: Ilene L
authors.initials: IL
authors.lastname: Hollin
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, NJ, USA.
authors.lastname: Caroline Young
authors.affiliation: Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
authors.forename: Caroline
authors.initials: C
authors.lastname: Hanson
authors.affiliation: Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
authors.forename: John F P
authors.initials: JFP
authors.lastname: Bridges
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, NJ, USA; RTI International, Research Triangle Park, NC, USA.
authors.forename: Holly
authors.initials: H
authors.lastname: Peay
mesh_terms: D020811:Biomedical Technology; D033161:Government Regulation; D006801:Humans; D007407:Interviews as Topic; D010358:Patient Participation; D057240:Patient Preference; D036301:Qualitative Research; D018570:Risk Assessment; D011795:Surveys and Questionnaires; D014481:United States; D014486:United States Food and Drug Administration
keywords: attribute development; community engagement; patient-centered benefit risk; stated preferences