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Public Workshops on Benefit-Risk Considerations

PDUFA VI Public Meeting: Characterizing FDA’s Approach to Benefit-Risk Assessment throughout the Medical Product Life Cycle

In accordance with commitments established in the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI) in 2017,[i] the FDA intends to publish draft guidance in 2020 on benefit-risk assessment for new drugs and biologics. The intent of the planned benefit-risk guidance is to provide drug sponsors and other stakeholders with better clarity about how considerations about a drug’s benefits, risks, and risk management factor into FDA’s regulatory decisions about its marketing authorization.

As part of our PDUFA VI commitments, FDA also committed to convening and/or participating in at least one meeting to gather industry, patient, researcher, and other stakeholder input on applying FDA’s Benefit-Risk Framework throughout the human drug development and post-marketing lifecycle and best approaches to communicating FDA’s benefit-risk assessment. Input from this meeting will support development of the draft guidance on benefit-risk assessment.

This meeting was convened on May 16, 2019, by the Duke-Margolis Center for Health Policy. The meeting focused on: 1) FDA’s planned benefit-risk assessment guidance; 2) activities that occur in pre-market development that best inform FDA’s benefit-risk assessment; 3) effectively communicating benefit-risk assessment information; and 4) using benefit-risk assessment to inform FDA and sponsor decision-making in the post-marketing setting.

More information about the meeting is available on the Duke-Margolis meeting page.


As part of our PDUFA V commitments to further develop and implement a structured approach to benefit-risk assessment in the human drug review process, FDA committed to holding two public workshops on benefit-risk considerations from the regulator’s perspective. Public Workshop I was held in 2014 (see below). Public Workshop II will be held on September 18, 2017.

Public Workshop II: Benefit-Risk Assessments in Drug Regulatory Decision-Making

The Food and Drug Administration (FDA or Agency) is holding this meeting to convene a discussion of topics related to the structured assessment of benefits and risks in drug regulatory decision-making. This meeting will focus on:

  • Regulatory and industry experiences with approaches to structured benefit-risk assessments
  • Approaches to incorporating patient perspectives into structured benefit-risk assessment
  • Exploration of methods to advance structured benefit-risk assessment.

The format of the meeting will include a series of presentations on the above topics related to structured assessment of benefits and risks, followed by a discussion on those topics with invited panelists and audience members. This workshop satisfies the second of FDA’s two workshop commitments in PDUFA V. This workshop is free and open to the public.

Date and Time:  September 18, 2017, 9:00 a.m. – 5:00 p.m.
Location: FDA White Oak Campus
10903 New Hampshire Avenue
Building 31, Great Room
Silver Spring, MD 20993
Meeting Information:
This web page will be updated as meeting materials and other information becomes available.
Meeting Slides:

Combined BR Meeting Slide Deck (PDF - 15 MB)

Webcast Link:


Meeting Agenda

Meeting Recording

Meeting Transcript (PDF - 1 MB)

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Public Workshop I: Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks

This two-day public workshop is being convened by the Institute of Medicine. The workshop will explore more systematic and structured approaches to evaluate and communicate:

  • the sources of uncertainty in the assessment of benefits and risks; and
  • their implications on human drug regulatory decisions.

Specifically, the workshop will explore potential analytical and communication approaches and identify key considerations on developing and incorporating potential uncertainty approaches into the assessment of benefits and risks. This workshop will consider the entire drug development lifecycle, including pre-market drug review and post-market safety surveillance.

The format of the meeting consists of a series of presentations by experts in various disciplines followed by a discussion of those issues from invited panelists and audience members. This workshop satisfies the first of FDA’s two workshop commitments in PDUFA V. This workshop is free and open to the public.

Federal Register Notice and Request for Comments disclaimer icon (The original comment due date was March 14, 2014. Due to meeting rescheduling, the comment period will be extended until June 11, 2014.) 


In its Draft Benefit-Risk Assessment Implementation Plan (PDF - 12MB), FDA identified as an area of further development the exploration of structured approaches to evaluate and communicate the uncertainty in the assessment of benefit or in the assessment of risks. FDA’s human drug regulatory decisions are informed by an extensive body of evidence on the safety and efficacy of a drug product.

In many cases, this evidence is subject to uncertainty arising from many sources. One example is the uncertainty in the degree to which pre-market clinical trial data translates to the post-market setting after the drug is approved and used in a much wider patient population. Another example is uncertainty about a potential safety signal that emerges in the post-market setting, where the basis for the finding comes from multiple evidence sources of varying levels of rigor.

Drawing conclusions in the face of uncertainty can be a complex and challenging task. Furthermore, being explicit about the impact of uncertainty on decision-making is an important part of communicating regulatory decisions.

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Related Information

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