FDA’s Benefit-Risk Framework for Human Drug Review
*Sara Eggers, FDA CDER  *Patrick Frey, FDA CDER 


For a human drug to be approved for marketing in the United States, the U.S. Food and Drug Administration must determine that the drug is effective for its intended use and that the benefits outweigh the risks of the product to the patient population. These decisions are informed by an extensive body of evidence about the product. They also take into account a number of other factors, including the nature and severity of the condition the drug is intended to treat or prevent, the availability of other therapies for the condition, and any risk management tools that might be necessary to ensure that the benefits of the drug outweigh its risks in the post-market setting. Since 2009, FDA has worked to develop a more systematic approach to support the assessment of benefits and risks and to be clearer and more consistent in communicating the reasoning behind drug regulatory decisions. FDA’s Benefit-Risk (B-R) Framework for human drug review is a structured qualitative approach supporting the identification and communication of the key issues, evidence, and uncertainties in FDA’s B-R assessment and how those considerations informed the regulatory decision. It makes explicit the therapeutic context in which benefits and risks were weighed. In the case of marketing approval, it provides rationale for any additional efforts necessary to further characterize the drug’s benefits or risks or to mitigate specific risks in the post-market setting. Further, the B-R Framework can be updated as more information becomes available in the post-market setting, reflecting the dynamic nature of B-R assessment throughout a drug’s lifecycle. This presentation will summarize the development of FDA’s B-R Framework for human drug products, its implementation into the drug review process, and opportunities for further development.