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 journalArticle

Abstract

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
Volume19
Issue number6
DOIs
StatePublished - Sep 1 2016

Fingerprint

Caregivers
Industry
Committee Membership
Duchenne Muscular Dystrophy
Advisory Committees
Research
Surveys and Questionnaires
Decision Making
Parents
Communication
Clinical Trials
Organizations
Interviews

Keywords

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

ASJC Scopus subject areas

  • Health Policy
  • Public Health, Environmental and Occupational Health

Cite this

Hollin, I. L., Caroline Young, Y., Hanson, C., Bridges, J. F. P., & Peay, H. (2016). Developing a Patient-Centered Benefit-Risk Survey: A Community-Engaged Process. Value in Health, 19(6), 751-757. https://doi.org/10.1016/j.jval.2016.02.014

Developing a Patient-Centered Benefit-Risk Survey : A Community-Engaged Process. / Hollin, Ilene L.; Caroline Young, Young; Hanson, Caroline; Bridges, John F P; Peay, Holly.

In: Value in Health, Vol. 19, No. 6, 01.09.2016, p. 751-757.

Research output: Contribution to journalArticle

Hollin, IL, Caroline Young, Y, Hanson, C, Bridges, JFP & Peay, H 2016, 'Developing a Patient-Centered Benefit-Risk Survey: A Community-Engaged Process', Value in Health, vol. 19, no. 6, pp. 751-757. https://doi.org/10.1016/j.jval.2016.02.014
Hollin IL, Caroline Young Y, Hanson C, Bridges JFP, Peay H. Developing a Patient-Centered Benefit-Risk Survey: A Community-Engaged Process. Value in Health. 2016 Sep 1;19(6):751-757. https://doi.org/10.1016/j.jval.2016.02.014
Hollin, Ilene L. ; Caroline Young, Young ; Hanson, Caroline ; Bridges, John F P ; Peay, Holly. / Developing a Patient-Centered Benefit-Risk Survey : A Community-Engaged Process. In: Value in Health. 2016 ; Vol. 19, No. 6. pp. 751-757.
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