Patient Centered Benefit-Risk Assessment (PCBR)

iStock_000014379683_ExtraSmallFramework Report Now Available

What is the issue?

Before approving a new medical device, regulators must evaluate its safety and effectiveness. One of the most important questions they ask is whether the clinical benefit of a device outweighs its risk. It makes sense to consider that question from the perspective of patients as well as researchers and clinicians. Patients and their families have a deep and personal understanding of what it is like to live with a disease, and they often have valuable insights on how a device could affect their quality of life. And in the end, patients are the ones who take the risks in order to achieve the benefits of medical therapies.

The U.S. Food and Drug Administration (FDA) has encouraged medical device companies to include patient perspectives on the benefits and risks of a new device when they seek approval to market it. But device manufacturers still need more specific guidelines on how to collect and use this data in the regulatory process.

What is the goal of this project?

MDIC and its partners are collaborating to improve our ability to include patient perspectives in the development, pre-market approval, and post-market evaluation of medical devices. First, we are trying to find scientifically valid ways to reliably assess patient views on the potential risks and benefits of specific devices. Second, we aim to establish a credible framework for incorporating that information into device development and benefit-risk assessments. We will share our findings with the FDA, which could then choose to use them in future guidance documents and regulatory decisions.

The FDA already recognizes the importance of patient preferences. In 2012, the agency’s Center for Devices and Radiological Health issued guidance for manufacturers on how it makes benefit-risk determinations during the pre-market review of certain medical devices. Significantly, the FDA emphasized that “patient tolerance of risk and perspective on benefits” is an important consideration. However, the FDA has not provided detailed instructions on how device developers should collect and use this information. For example, what types of patient preferences should be measured? What are the best ways to measure those preferences? How and when should manufacturers provide this data to regulators? These are some of the questions that MDIC intends to address. CDRH issues draft guidance in May 2015 on patient preference information in submission, review in PMAs, HDE Application and De Novo requests and inclusion in device labeling.

What has the project produced?

  • A framework for incorporating information on those preferences into benefit-risk assessments of new medical technology.
  • A catalog of methods that can be used to assess patient preferences about the benefits and risks of a medical technology.
  • An analysis of gaps in current methods for assessing patient preferences.
  • An agenda for further research. Download report here.

Who is working on this project?

MDIC has assembled a steering committee of member organization and FDA representatives, patient advocates, and other experts to guide work on this project.