Incorporating patient-preference evidence into regulatory decision making
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Patients have a unique role in deciding what treatments should be available for them and regulatory agencies should take their preferences into account when making treatment approval decisions. This is the first study designed to obtain quantitative patient-preference evidence to inform regulatory approval decisions by the Food and Drug Administration Center for Devices and Radiological Health.
Five-hundred and forty United States adults with body mass index (BMI) ≥30 kg/m2 evaluated tradeoffs among effectiveness, safety, and other attributes of weight-loss devices in a scientific survey. Discrete-choice experiments were used to quantify the importance of safety, effectiveness, and other attributes of weight-loss devices to obese respondents. A tool based on these measures is being used to inform benefit-risk assessments for premarket approval of medical devices.
Respondent choices yielded preference scores indicating their relative value for attributes of weight-loss devices in this study. We developed a tool to estimate the minimum weight loss acceptable by a patient to receive a device with a given risk profile and the maximum mortality risk tolerable in exchange for a given weight loss. For example, to accept a device with 0.01 % mortality risk, a risk tolerant patient will require about 10 % total body weight loss lasting 5 years.
Patient preference evidence was used make regulatory decision making more patient-centered. In addition, we captured the heterogeneity of patient preferences allowing market approval of effective devices for risk tolerant patients. CDRH is using the study tool to define minimum clinical effectiveness to evaluate new weight-loss devices. The methods presented can be applied to a wide variety of medical products. This study supports the ongoing development of a guidance document on incorporating patient preferences into medical-device premarket approval decisions.
KeywordsPatient preferences Weight-loss devices Obesity treatment FDA Benefit-risk assessment Regulatory-approval decisions
We acknowledge Jeffrey Shuren, MD, JD for his support of the study and insightful advice on the manuscript. We also acknowledge Priya Venkataraman-Rao, MD; Megan Shackelford, MS; Rebecca Nipper; Richard Kotz, MS; Kathleen Olvey; and Martin Golding, MD, for their regulatory input in the development of the survey instrument. We are grateful to the FDA CDRH Obesity Devices Working Group for their comments on interpretation of study results and their feedback on using the MinB–MaxR calculator in regulatory reviews of weight-loss device submissions. The members of that group include Jeffrey Cooper, DVM; Megan Shackelford, MS; Irene Bacalocostantis, PhD; Brandan Reid, PhD; Martha Betz, PhD; Elizabeth Katz, PhD; David Pudwill; Martin Golding, MD; Priya Venkataraman-Rao, MD; and Benjamin Fisher, PhD. The acknowledged persons above are of CDRH, FDA.
Drs. Hauber and Gonzalez received compensation for their work through a contract with the Center for Devices and Radiological Health (CDRH) of the U.S. Food and Drug Administration (FDA). Ho, Lerner, Neuland, Whang, McMurry-Heath, and Irony have no conflicts of interest or financial ties to disclose.
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