Variation Among Patients With Crohn's Disease in Benefit vs Risk Preferences and Remission Time Equivalents.
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2019-05-14
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BACKGROUND & AIMS:Patients with Crohn's disease (CD) must make decisions about their treatment. We aimed to quantify patients' preferences for different treatment outcomes and adverse events. We also evaluated the effects of latent class heterogeneity on these preferences. METHODS:An online stated-preference survey was completed by 812 individuals with CD in the Crohn's and Colitis Foundation Partners cohort (IBD Partners). Patients were given information on symptoms and severity of active disease; duration of therapy with corticosteroids; and risks of serious infection, cancer and surgery. Patients were asked to assume that their treatment was not working and to choose an alternative therapy. The primary outcome was remission-time equivalents (RTE) of a given duration of symptom severity or treatment-related risk. Latent class choice models identified groups of patients with dominant treatment-outcome preferences and associated patient characteristics with these groups. RESULTS:Latent class analysis demonstrated 3 distinct groups of survey responders whose choices were strongly influenced by avoidance of active symptoms (61%), avoidance of corticosteroid use (25%), or avoidance of risks of cancer, infection or surgery (14%) when choosing a therapy. Class membership was correlated with age, sex, mean short CD activity index score and corticosteroid avoidance. RTEs in each latent class differed significantly from the mean RTEs for the overall sample, although the symptom-avoidant class most closely approximated the overall sample. CONCLUSIONS:In an online survey of patients with CD, we found substantial heterogeneity in preference for medication efficacy and risk of harm. Physicians and regulators should therefore not assume that all patients have mean-value preferences-this could result in significant differences in health-technology assessment models.
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Bewtra, Meenakshi, Shelby D Reed, F Reed Johnson, Frank I Scott, Erin Gilroy, Robert S Sandler, Wenli Chen, James D Lewis, et al. (2019). Variation Among Patients With Crohn's Disease in Benefit vs Risk Preferences and Remission Time Equivalents. Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association. 10.1016/j.cgh.2019.05.010 Retrieved from https://hdl.handle.net/10161/19117.
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Shelby Derene Reed
Shelby D. Reed, PhD, is Professor in the Departments of Population Health Sciences and Medicine at Duke University’s School of Medicine. She is the director of the Center for Informing Health Decisions and Therapeutic Area leader for Population Health Sciences at the Duke Clinical Research Institute (DCRI). She also is core faculty at the Duke-Margolis Center for Health Policy. Dr. Reed has over 20 years of experience leading multidisciplinary health outcomes research studies. Dr. Reed has extensive expertise in designing and conducting trial-based and model-based cost-effectiveness analyses of diagnostics, drugs and patient-centered interventions. In 2016, she co-founded the Preference Evaluation Research (PrefER) Group at the DCRI, and she currently serves as its director. She and the group are frequently sought to conduct stated-preference studies to inform regulatory decisions, health policy, care delivery, value assessment and clinical decision making with applied projects spanning a wide range of therapeutic areas. She served as President for ISPOR in 2017-2018, and she currently is Past-Chair of the Society’s Health Science Policy Council.
Areas of expertise: Health Economics, Health Measurement, Stated Preference Research, Health Policy, and Health Services Research
F. Reed Johnson
F. Reed Johnson, PhD, has more than 40 years of academic and research experience in health and environmental economics. He has served on the faculties of several universities in the United States, Canada, and Sweden, and as Distinguished Fellow at Research Triangle Institute. He currently is Senior Research Scholar in the Duke Clinical Research Institute. As a staff member in the US Environmental Protection Agency’s environmental economics research program during the 1980s, Reed helped pioneer the development of basic non-market valuation techniques which are widely used for benefit-cost analysis in health and environmental economics. He has designed and analyzed numerous surveys for measuring preferences for and value of health outcomes, health risk reductions, and improved environmental quality.
Dr. Johnson has over 140 publications in books and peer-reviewed journals. His research has been published in various medical journals, the Review of Economics and Statistics, Journal of Health Economics, Medical Decision Making, Health Economics, Value in Health, Journal of Policy Analysis and Management, and other journals. He has coauthored a book on techniques for using existing environmental and health value estimates for policy analysis.
His current research involves quantifying patients’ willingness to accept sideeffect risks in return for therapeutic benefits and estimating general time equivalences among health states. He led the first FDA sponsored study on patients’ willingness to accept benefit-risk tradeoffs for new health technologies. The study was used to develop recent FDA guidance on submitting patient-preference data to support regulatory reviews of medical devices.
Areas of expertise: Clinical Decision Sciences, Health Measurement, Health Policy, and Health Economics
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