FDA issues final Guidance on Benefit-Risk Assessment

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 

  • collected by any persons (including patients, family members and caregivers of patients, patient advocacy organizations, disease research foundations, researchers, and drug manufacturers),
  • intended to provide information about patients’ experiences with a disease or condition (including the impact of such disease or condition, or a related therapy on patients’ lives) and patient preferences with respect to treatment of such disease or condition.

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

FDA's benefit-risk assessment comprises a case-specific, multi-disciplinary assessment of science and medicine, which considers the following:

  • The therapeutic context in which the drug will be used.
  • The evidence submitted in the premarket application and/or generated in the postmarket setting (e.g. from clinical data, non-clinical data, patient experience data, product quality information, spontaneous reports of adverse events, and epidemiologic data).
    Such data may have been collected specifically to address questions regarding the benefits and risks of the drug or routinely collected from a variety of real-world-data sources.
  • The uncertainties about the drug’s benefits and risks.
  • FDA’s regulatory options to manage risks and to further reduce uncertainties (e.g. via product labeling, REMS, additional clinical studies).

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.

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