Caregiver Preferences for Emerging Duchenne Muscular Dystrophy Treatments: A Comparison of Best-Worst Scaling and Conjoint Analysis
- 390 Downloads
Through Patient-Focused Drug Development, the US Food and Drug Administration (FDA) documents the perspective of patients and caregivers and are currently conducting 20 public meetings on a limited number of disease areas. Parent Project Muscular Dystrophy (PPMD), an advocacy organization for Duchenne muscular dystrophy (DMD), has demonstrated a community-engaged program of preference research that would complement the FDA’s approach.
Our objective was to compare two stated-preference methods, best-worst scaling (BWS) and conjoint analysis, within a study measuring caregivers’ DMD-treatment preferences.
Within one survey, two preference-elicitation methods were applied to 18 potential treatments incorporating six attributes and three levels. For each treatment profile, caregivers identified the best and worst feature and intention to use the treatment. We conducted three analyses to compare the elicitation methods using parameter estimates, conditional attribute importance and policy simulations focused on the 18 treatment profiles. For each, concordance between the results was compared using Spearman’s rho.
BWS and conjoint analysis produced similar parameter estimates (p < 0.01); conditional attribute importance (p < 0.01); and policy simulations (p < 0.01). Greatest concordance was observed for the benefit and risk parameters, with differences observed for nausea and knowledge about the drug—where a lack of monotonicity was observed when using conjoint analysis.
The observed concordance between approaches demonstrates the reliability of the stated-preference methods. Given the simplicity of combining BWS and conjoint analysis on single profiles, a combination approach is easily adopted. Minor irregularities for the conjoint-analysis results could not be explained by additional analyses and needs to be the focus of future research.
KeywordsDuchenne Muscular Dystrophy Conjoint Analysis Duchenne Muscular Dystrophy Treatment Profile Elicitation Format
The authors appreciate the leadership and commitment of the Parent Project Muscular Dystrophy oversight committee: Pat Furlong, Brian Denger, Sharon Hesterlee, and Kathi Kinnett. We are indebted to the stakeholder informants, parents who participated in the cognitive interviews, and caregivers who completed the survey. This paper was presented at the first meeting of the International Academy for Health Preference Research (IAHPR), 8 November 2014. We are grateful for the feedback we received from the participants of this meeting and of the peer reviewers of this manuscript. This study was conducted with the support from PPMD. Dr. Bridges also acknowledges support from the Patient-Centered Outcomes Research Institute (PCORI) Methods Program Award (ME-1303-5946). ILH conducted data analyses and wrote the manuscript. JFPB and HP conceived and designed the study, analysis plan, and assisted in the writing and reviewing of the manuscript. All authors reviewed and approved the final draft of this manuscript. JFPB acts as the overall guarantor of this article.
Conflict of interest
Holly Peay is an employee of PPMD and John Bridges was hired as a consultant by PPMD to provide methodological expertise. The authors have no conflicts to disclose.
- 3.Center for Disease Control and Prevention (CDC). Prevalence of Duchenne/Becker muscular dystrophy among males aged 5–24 years—Four states, 2007. MMWR Morb Wkly Rep. 2009; 58:1119–22.Google Scholar
- 8.Ouyang L, Grosse SD, Kenneson A. Health care utilization and expenditures for children and young adults with muscular dystrophy in a privately insured population. J Child Neurol. 2008;23(8):883-8.Google Scholar
- 11.Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids 2–4 April 2004, Naarden, The Netherlands. Neuromuscul Disord. 2004;14(8):526–34.PubMedCrossRefGoogle Scholar
- 14.Parent Project Muscular Dystrophy. Putting Patients First: Recommendations to speed responsible access to new therapies for Duchenne muscular dystrophy and other rare, serious and life-threatening neurologic disorders. Hackensack; 2013.Google Scholar
- 15.Parent Project Muscular Dystrophy. Guidance for Industry: Duchenne Muscular Dystrophy Developing Drugs for Treatment over the Spectrum of Disease. Hackensack; 2014.Google Scholar
- 16.U.S. Food and Drug Administration. Enhancing Benefit-Risk Assessment in Regulatory Decision-Making. Silver Spring; 2014. http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm. Accessed June 26 2014.
- 18.“Prescription Drug User Fee Act Patient-Focused Drug Development; Announcement of Disease Areas for Meetings Conducted in Fiscal Years 2013–2015”. Federal Register 2013; 78:21613–4.Google Scholar
- 27.Finn A, Louviere JJ. Determining the appropriate response to evidence of public concern: the case of food safety. J Public Policy Market. 1992:12–25.Google Scholar
- 36.Cohen S, Orme B. What’s your preference? Market Res. 2004;16(2):32–7.Google Scholar
- 37.Cohen S, editor. Maximum difference scaling: improved measures of importance and preference for segmentation. Sawtooth Software Conference Proceedings, Sawtooth Software, Inc; 2003.Google Scholar
- 41.Kuhfeld W. Orthogonal arrays [TS-723]. Cary: SAS [online]. 2010.Google Scholar
- 44.Reed Johnson F, Lancsar E, Marshall D, Kilambi V, Mühlbacher A, Regier DA et al. Constructing experimental designs for discrete-choice experiments: report of the ISPOR conjoint analysis experimental design good research practices task force. Value Health. 2013;16(1):3–13.Google Scholar
- 45.Carson RT, Hanemann WM, Kopp RJ, Jon AK, Robert Cameron M, Presser S et al. Referendum design and contingent valuation: the NOAA panel’s no-vote recommendation. Rev Econ Stat. 1998;80(2):335–8.Google Scholar
- 47.McFadden D. Conditional logit analysis of qualitative choice behavior. In: Zarembka P, editor. Frontiers in econometrics. New York: Academic Press; 1974. p. 105–42.Google Scholar