Project: Patient Centered Benefit-Risk

The MDIC Patient Centered Benefit-Risk (PCBR) Project grew out of emphasis by FDA’s Center for Devices and Radiological Health (CDRH) on benefit-risk assessment as a central component of the medical device regulatory decision-making process.

MDIC catalyzed a multi-stakeholder project to help the medical device community evaluate when and how to communicate benefit, risk, and uncertainty information.

Full Summary

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.

Given that there were no widely accepted approaches for assessing patient preferences that have been used in the regulatory process to date, MDIC identified the opportunity to help the medical device community evaluate when and how patient preference information might be incorporated into regulatory decision making. To oversee this project, 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 Framework report.

The Steering Committee divided the PCBR Project 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 in the Framework Report. 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 FDA through a BAA contract (HHSF223201400011C) made this Framework Report possible and was primarily used to fund the work of RTI and outside experts on the Catalog.
Articles in the

Patient Centered Benefit-Risk (PCBR)

Patient Preference Study Design Guide – Qualitative Steps: First steps for sponsors initiating a patient preference study

Patient Preference Study Design Guide – Qualitative Steps: First steps for sponsors initiating a patient preference study

This study design guide provides an outline of the qualitative steps for initiating a patient preference study. The guide only includes qualitative steps used to develop the preference survey and does not include the development of the preference study protocol or...

A Framework for Incorporating Information on Patient Preferences Regarding Benefit and Risk into Regulatory Assessments of New Medical Technology

A Framework for Incorporating Information on Patient Preferences Regarding Benefit and Risk into Regulatory Assessments of New Medical Technology

The Patient Centered Benefit-Risk (PCBR) Framework introduces the concept of the total product development lifecycle and discusses how patient preference information might be useful at each stage of this lifecycle. The report provides background on the concepts of...