Characterizing the FDA’s Approach to Benefit-Risk Assessment throughout the Medical Product Life Cycle

Project Report

Characterizing the FDA’s Approach to Benefit-Risk Assessment throughout the Medical Product Life Cycle

With the sixth reauthorization of the Prescription Drug User Fee Act (PDFUA VI), the U.S. Food and Drug Administration (FDA or Agency) has committed to furthering its implementation of structured benefit-risk assessment (BRA) in medical product review and enhancing the integration of patient input in regulatory decision-making, according to the Agency’s PDUFA VI implementation plan.[i]  As part of this commitment, the Agency will issue draft guidance on its approach to BRA. This guidance will articulate, in part, how key benefit-risk considerations about a drug’s benefits, risks, and risk management options inform BRA and how the Agency’s benefit-risk framework (BRF) supports and structures decision-making related to market authorizations for medical products. Additionally, the guidance will discuss how relevant patient experience data and related information may be used to inform BRA. As part of its PDUFA VI implementation plan, the Agency also committed to collect and consider stakeholder input on approaches to communicating BRA information to biopharmaceutical industry stakeholders (developers) and the public.[ii]

Accordingly, under a cooperative agreement with the FDA, the Duke-Margolis Center for Health Policy (Margolis Center) convened a public meeting in May 2019 to facilitate discussion on the implementation and communication of the FDA’s BRF. Following the public meeting, the Margolis Center sought to address additional questions related to benefit-risk approaches and convened a follow-on teleconference. Varied industry stakeholders participated, as well as Agency colleagues involved in planning the public meeting. The follow-on teleconference focused on industry stakeholders’ decision-making related to overall BRA, internal benefit-risk communication practices, and approaches to communicating benefit-risk information with the Agency. The following is a summary of considerations and recommendations discussed at these convenings that the Agency can consider during guidance development and the ongoing implementation of the BRF. This summary describes input from a broad range stakeholders who attended the public meeting, including regulatory, industry, patient and academic stakeholders, as well as input from industry stakeholders who participated in the follow-on teleconference.[1]


[1] Stakeholder input has been summarized but does not necessarily reflect consensus views among public meeting and follow-on teleconference participants, nor official positions of the U.S. Food and Drug Administration (FDA).

[i] U.S. Food and Drug Administration. Benefit-Risk Assessment in Drug Regulatory Decision-Making: Draft PDUFA VI Implementation Plan (FY 2018-2022). Published March 30, 2018. Accessed June 26, 2019.

[ii] Ibid [at Draft PDUFA VI Implementation Plan]

Duke-Margolis Authors