Surgical Endoscopy

, Volume 29, Issue 10, pp 2984–2993 | Cite as

Incorporating patient-preference evidence into regulatory decision making

  • Martin P. Ho
  • Juan Marcos Gonzalez
  • Herbert P. Lerner
  • Carolyn Y. Neuland
  • Joyce M. Whang
  • Michelle McMurry-Heath
  • A. Brett Hauber
  • Telba Irony
Article

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/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.

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.

Keywords

Patient preferences Weight-loss devices Obesity treatment FDA Benefit-risk assessment Regulatory-approval decisions 

Supplementary material

464_2014_4044_MOESM1_ESM.docx (148 kb)
Supplementary material 1 (DOCX 148 kb)

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Copyright information

© Springer Science+Business Media New York (outside the USA) 2015

Authors and Affiliations

  • Martin P. Ho
    • 1
  • Juan Marcos Gonzalez
    • 2
  • Herbert P. Lerner
    • 1
  • Carolyn Y. Neuland
    • 1
  • Joyce M. Whang
    • 1
  • Michelle McMurry-Heath
    • 1
    • 3
  • A. Brett Hauber
    • 2
  • Telba Irony
    • 1
  1. 1.Center for Devices and Radiological HealthU.S. Food and Drug AdministrationSilver SpringUSA
  2. 2.RTI Health SolutionsDurhamUSA
  3. 3.FaegreBD ConsultingWashingtonUSA

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