TY - JOUR
T1 - Developing a Patient-Centered Benefit-Risk Survey
T2 - A Community-Engaged Process
AU - Hollin, Ilene L.
AU - Caroline Young, Young
AU - Hanson, Caroline
AU - Bridges, John F.P.
AU - Peay, Holly
N1 - Funding Information:
Source of financial support: I.L. Hollin, C. Hanson, and J.F.P. Bridges received support from a grant (#01212) from Parent Project Muscular Dystrophy (PPMD). PPMD received funding for this project from Santhera Pharmaceuticals. J.F.P. Bridges also received support from a Patient-Centered Outcomes Research Institute (PCORI) Methods Program Award (ME-1303-5946) and through the Johns Hopkins-FDA Center for Excellence in Regulatory Science and Innovation (CERSI) (1U01FD004977-01).
Publisher Copyright:
© 2016
PY - 2016/9/1
Y1 - 2016/9/1
N2 - 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.
AB - 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.
KW - attribute development
KW - community engagement
KW - patient-centered benefit risk
KW - stated preferences
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U2 - 10.1016/j.jval.2016.02.014
DO - 10.1016/j.jval.2016.02.014
M3 - Article
C2 - 27712702
AN - SCOPUS:84992396758
SN - 1098-3015
VL - 19
SP - 751
EP - 757
JO - Value in Health
JF - Value in Health
IS - 6
ER -