The MDIC Patient Centered Benefit-Risk (PCBR) Project grew out of FDA’s Centers for Devices and Radiological Health (CDRH) emphasis on benefit-risk assessment as a central component of the medical device approval process. CDRH’s 2012 “Guidance for Industry and Food and Drug Administration Staff Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications” discusses the importance of bringing the patient’s perspective into CDRH benefit-risk assessments, and recommends that sponsors interact with FDA staff regarding the development of patient centered benefit-risk information. However, the document does not discuss in what situations or how sponsors should collect and present that information.
Given that there are no widely accepted approaches for assessing patient preferences that have been used in the regulatory process to date, MDIC saw an opportunity to help industry and the FDA think about when and how patient preference information might be incorporated into regulatory decision making. To oversee this project, the MDIC set up a PCBR Steering Committee consisting of interested participants among MDIC’s member organizations supplemented with experts in decision science and preference assessment methods from academia and other organizations. The members of the PCBR Steering Committee are listed in Exhibit 1-2 of the report.
The Steering Committee divided the PCBR Project down into two complementary efforts:
- The development of a “framework” for incorporating patient preference information into benefit-risk assessment, which became this “Framework Report;” and
- The development of a “catalog of methods” that can be used to collect and analyze patient preference information, which is the “Catalog of Methods” included as Appendix A. The MDIC PCBR Steering Committee contracted the development of the Catalog of Methods to RTI Health Solutions with the assistance of a “Catalog Working Group” that included experts in a variety of methods related to assessing patient preferences, as listed in Exhibit 1-4. Financial support from a FDA BAA contract (HHSF223201400011C) made this Framework Report possible, and was primarily used to fund the work of RTI and outside experts on the Catalog.
Introducing the Framework for Integrating Patient Perspective into Medical Device Benefit-Risk Assessments
In 2015, MDIC held a public event for the release of its Framework & Catalog of Methods for ‘Incorporating Information on Patient Preferences Regarding Benefit and Risk into the Regulatory Assessments of New Medical Technologies’. As a result of CDRH’s 2012 Guidance that discusses the importance of bringing the patient’s perspective into the CDRH benefit-risk assessments, MDIC brought together a steering committee with members from the medical device industry, government and patient centered non-profits to develop a framework for how sponsors and regulators might incorporate the patient perspective on benefit and risk. This Framework provides background on the concepts of benefit-risk and patient preference, discusses the potential value of including patient benefit-risk in a regulatory submission and when in the product lifecycle such information might be collected, outlines factors to consider when selecting a patient preference method, and discusses considerations regarding the use of patient preference information in the regulatory process.
Bill Murray, President and CEO, MDIC
Jeff Shuren, Director, FDA Center for Devices and Radiological Health
Ross Jaffe, Versant Ventures & National Venture Capital Association;
Board Champion for MDIC Patient Centered Benefit-Risk Assessment
Brett Hauber, RTI Health Solutions
Framework Section II: Bennett Levitan, Johnson & Johnson
Framework Section III: Diana Salditt, Medtronic
Framework Section V: Brett Hauber, RTI Health Solutions
Framework Section VI: Telba Irony, FDA
Discussion panel: Brett Hauber, Telba Irony, Bennett Levitan, Bryan Olin, Diana Salditt
Discussion moderator: Bill Murray
Discussion panel: Bob Becker, Martin Ho, Telba Irony, Kathryn O’Callaghan, Anindita Saha
Discussion moderator: Kim McCleary, Fastercures
Stephanie Christopher, MDIC
Discussion panel: Marc Boutin, Jessica Foley, Kim McCleary, Bray Patrick-Lake
Discussion moderator: Kathryn O’Callaghan
Discussion panel: Brett Hauber, Martin Ho, Bennett Levitan, Bryan Luce
Discussion moderator: Bill Murray
Dr. Robert Califf, FDA