Developing a Patient-Centered Benefit-Risk Survey: A Community-Engaged Process

Ilene L. Hollin, Young Caroline Young, Caroline Hanson, John F.P. Bridges, Holly Peay

Research output: Contribution to journalArticlepeer-review

30 Scopus citations


Objectives 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.

Original languageEnglish (US)
Pages (from-to)751-757
Number of pages7
JournalValue in Health
Issue number6
StatePublished - Sep 1 2016


  • attribute development
  • community engagement
  • patient-centered benefit risk
  • stated preferences

ASJC Scopus subject areas

  • Health Policy
  • Public Health, Environmental and Occupational Health


Dive into the research topics of 'Developing a Patient-Centered Benefit-Risk Survey: A Community-Engaged Process'. Together they form a unique fingerprint.

Cite this