title: Incorporating patient-preference evidence into regulatory decision making.
abstract: BACKGROUND
  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.
  
  
  METHODS
  Five-hundred and forty United States adults with body mass index (BMI) ≥ 30 kg/m(2) 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.
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Center for Devices and Radiological Health, U.S. Food and Drug Administration, 10903 New Hampshire Ave, Building 66, Room 2232, Silver Spring, MD, 20993-0002, USA. martin.ho@fda.hhs.gov.
authors.forename: Martin P
authors.initials: MP
authors.lastname: Ho
authors.affiliation: RTI Health Solutions, Durham, USA. jgonzalez@rti.org.
authors.forename: Juan Marcos
authors.initials: JM
authors.lastname: Gonzalez
authors.affiliation: Center for Devices and Radiological Health, U.S. Food and Drug Administration, 10903 New Hampshire Ave, Building 66, Room 2232, Silver Spring, MD, 20993-0002, USA. herbert.lerner@fda.hhs.gov.
authors.forename: Herbert P
authors.initials: HP
authors.lastname: Lerner
authors.affiliation: Center for Devices and Radiological Health, U.S. Food and Drug Administration, 10903 New Hampshire Ave, Building 66, Room 2232, Silver Spring, MD, 20993-0002, USA. carolyn.neuland@fda.hhs.gov.
authors.forename: Carolyn Y
authors.initials: CY
authors.lastname: Neuland
authors.affiliation: Center for Devices and Radiological Health, U.S. Food and Drug Administration, 10903 New Hampshire Ave, Building 66, Room 2232, Silver Spring, MD, 20993-0002, USA. joyce.whang@fda.hhs.gov.
authors.forename: Joyce M
authors.initials: JM
authors.lastname: Whang
authors.affiliation: Center for Devices and Radiological Health, U.S. Food and Drug Administration, 10903 New Hampshire Ave, Building 66, Room 2232, Silver Spring, MD, 20993-0002, USA. michelle.mcmurry-heath@fda.hhs.gov.|FaegreBD Consulting, Washington, USA. michelle.mcmurry-heath@fda.hhs.gov.
authors.forename: Michelle
authors.initials: M
authors.lastname: McMurry-Heath
authors.affiliation: RTI Health Solutions, Durham, USA. abhauber@rti.org.
authors.forename: A Brett
authors.initials: AB
authors.lastname: Hauber
authors.affiliation: Center for Devices and Radiological Health, U.S. Food and Drug Administration, 10903 New Hampshire Ave, Building 66, Room 2232, Silver Spring, MD, 20993-0002, USA. telba.irony@fda.hhs.gov.
authors.forename: Telba
authors.initials: T
authors.lastname: Irony
mesh_terms: D050110:Bariatric Surgery / Q000295:instrumentation*; D002755:Choice Behavior; D003430:Cross-Sectional Studies; D003657:Decision Making; D033161:Government Regulation; D006801:Humans; D009765:Obesity / Q000601:surgery; D057240:Patient Preference; D018570:Risk Assessment; D011795:Surveys and Questionnaires; D014481:United States; D014486:United States Food and Drug Administration
keywords: Benefit-risk assessment; FDA; Obesity treatment; Patient preferences; Regulatory-approval decisions; Weight-loss devices