For Industry

Public Workshops on Benefit-Risk Considerations

Due to the inclement weather on Feb. 13, 2014, the second day of this workshop was postponed until May 12, 2014. The comment period for the public docket will be extended until June 11, 2014. More information will be published in a forthcoming Federal Register Notice. We will update this webpage as information becomes available.

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 will be held on February 12-13, 2014 (see below). Public Workshop II will be held at a later date in PDUFA V.

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.

Date and Time: Day 1: February 12, 2014, 9:00 a.m. – 5:00 p.m.
Day 2: NEW DATE: May 12, 2014: 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:
More information, including the meeting agenda can be found at the IOM meeting web page disclaimer icon . This web page will be updated as meeting materials and other information becomes available.
Online registration for this workshop has closed; however, registration will be available in-person at the workshop on May 12.

Webcast Recordings

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 - 1MB), 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.


Page Last Updated: 12/11/2014
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