Incorporating patient-preference evidence into regulatory decision making

Area of Science:

• Medical device regulation
• Patient-centered healthcare
• Health economics

Background:

• Patient preferences are crucial for medical device availability and regulatory approval.
• Regulatory agencies like the Food and Drug Administration (FDA) Center for Devices and Radiological Health (CDRH) are increasingly considering patient input.
• This study is the first to gather quantitative patient-preference data for informing regulatory decisions.

Purpose of the Study:

• To quantify patient preferences for attributes of weight-loss devices.
• To inform benefit-risk assessments for premarket approval of medical devices.
• To make regulatory decision-making more patient-centered.

Main Methods:

• A scientific survey of 540 US adults with a body mass index (BMI) ≥ 30 kg/m².
• Discrete-choice experiments to evaluate tradeoffs between device effectiveness, safety, and other attributes.
• Development of a tool to estimate patient-acceptable weight loss and mortality risk.

Main Results:

• Patient preference scores were derived from choices, indicating the relative value of different device attributes.
• A tool was created to determine minimum acceptable weight loss for a given risk profile and maximum tolerable mortality risk for a given weight loss.
• For example, a risk-tolerant patient may accept a 0.01% mortality risk for approximately 10% total body weight loss over 5 years.

Conclusions:

• Patient preference evidence enhances regulatory decision-making, making it more patient-centered.
• The study captured patient preference heterogeneity, enabling market approval for effective devices for risk-tolerant individuals.
• The developed tool is used by CDRH to define minimum clinical effectiveness for new weight-loss devices, with potential application to other medical products.