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  1. Prescription Drug User Fee Amendments

Enhancing Benefit-Risk Assessment in Regulatory Decision-Making

Benefit-risk assessment is an integral part of FDA’s regulatory review of marketing applications for new drugs and biologics. These assessments capture the Agency’s evidence, uncertainties, and reasoning used to arrive at its final determination for specific regulatory decisions. Additionally, they serve as a tool for communicating this information to those who wish to better understand FDA’s thinking.

Beginning with commitments made as part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), the Agency has identified and implemented enhancements with the goal of increasing the clarity, transparency, and consistency of FDA’s benefit-risk assessments in human drug review. With passage of the 21st Century Cures Act, FDA also has an imperative to consider how relevant patient experience data and related information can be incorporated into the structured benefit-risk assessment framework to inform regulatory decision-making. 

As part of its commitments under PDUFA VI and requirements under 21st Century Cures, FDA has developed a draft guidance for industry on benefit-risk assessment for new drug and biological products. The intent of this guidance, when finalized, is to clarify for drug  sponsors and other stakeholders how considerations about a drug’s benefits, risks, and risk management options factor into certain premarket and postmarket regulatory decisions that the Agency makes about new drug applications submitted under section 505(c) of the Federal Food, Drug, and Cosmetic Act as well as biologics license applications submitted under section 351(a) of the Public Health Service Act.

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 Input from this meeting supported development of the draft guidance on benefit-risk assessment.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.

Additional information related to FDA’s implementation and ongoing enhancement of benefit-risk assessment in human drug and biologic regulatory decision-making is available below.



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