Keywords: benefit-risk, PDUFA, FDA
Benefit-risk assessment in FDA’s regulatory context is a judgment as to whether the expected benefits (with their uncertainties) of the drug outweigh the potential risks (with their uncertainties and options to manage risks), associated with its expected use. The assessment of a drug’s benefits versus risks along that product’s lifecycle involves consideration of a diverse set of factors related to the condition, medical need, strength of evidence, risk mitigation options, statutory requirements, real-world use. Evaluation of these considerations requires a diverse review team with expertise in many areas — statistical analysis being a critical component. In 2009, FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) began development of a structured, qualitative approach to benefit-risk assessment in human drug and biologic regulatory review, with the objective of increasing the clarity and transparency of decision making at the Agency. As of 2017, FDA has implemented the resulting Benefit-Risk Framework into its review processes. Under PDUFA VI, FDA has committed to continue to enhance the Framework and its implementation. A planned draft guidance will articulate FDA’s approach to benefit-risk assessment and interactions between sponsors and FDA, and FDA and the public, on benefit-risk assessment. In addition, the Agency has begun to explore more advanced benefit-risk assessment techniques that can supplement use of the Framework. This presentation will discuss the current state of benefit-risk assessment for human drugs and biologics at FDA and plans for future enhancement and implementation. Conclusions from a 2019 public meeting to inform guidance for industry will also be discussed.