19/20 March 2024
The U.S. Food and Drug Administration (FDA) published the final guidance on Benefit-Risk Assessment. According to the agency, the intent of the guidance is to clarify how considerations about a drug’s benefits, risks, and risk management options factor into certain premarket and postmarket regulatory decisions. The document first provides important considerations that flow into the Center for Drug Evaluation and Research’s (CDER) and the Center for Biologics Evaluation and Research’s (CBER) benefit-risk assessments, including how patient experience data can be used. According to the guideline, patient experience data is
The guidance also discusses how sponsors can inform FDA’s benefit-risk assessment through the design and conduct of a development program, as well as how they may present benefit and risk information in the marketing application. In addition, opportunities for interaction between the FDA and sponsors to discuss benefit-risk considerations in connection with the development of a new drug application (NDA) as well as biologics license application (BLA) are provided. The document then concludes with additional considerations on benefit-risk assessments in the postmarket setting.
FDA's benefit-risk assessment comprises a case-specific, multi-disciplinary assessment of science and medicine, which considers the following:
In any case, sponsors, should not wait for a periodic safety update (PSUR) to report a potentially serious safety concern. New information about a potential serious safety concern that could have an impact on a drug’s benefit-risk profile should be communicated promptly to the FDA.
For more information please see the final FDA Guidance on Benefit-Risk Assessment for New Drug and Biological Products.