FDA Proposes Benefit-Risk Assessment Guidance for New Drug and Biological Products - Celegence

FDA Proposes Benefit-Risk Assessment Guidance for New Drug and Biological Products

In October 2023, the FDA introduced a significant guidance document, “Benefit-risk assessment for new drug and biological products”, for the pharmaceutical industry. This guidance focuses on the assessment of benefit-risk for new drugs and biological products. This comprehensive guideline aims to bring clarity for drug sponsors and stakeholders regarding how the FDA considerations (drug’s benefits, risks, and risk management options) in premarket and post-market regulatory decisions. This guidance pertains to new drug applications (NDAs) under section 505(b) of the Federal Food, Drug, and Cosmetic Act and biologics license applications (BLAs) under section 351(a) of the Public Health Service Act.

Key Elements of the Guideline

This guidance addresses the following key points for Regulatory decisions for approval:

  • Labeling considerations (including boxed warnings)
  • Post-marketing studies and commitments
  • Risk evaluation and mitigation strategies

Note: The guidance document does not cover decisions during the drug development lifecycle, such as those related to first-in-human trials or expanded access applications.

About the Content in Focus

The FDA’s approach to benefit-risk assessment of new drugs and biological products plays a pivotal role in the FDA’s regulatory review for marketing applications. The document provided in-depth details regarding the FDA’s benefit-risk framework and other important considerations.

  • The following are the key points for the assessment of benefit-risk assessment of the drug as per the FDA:
    • Therapeutic context of the drug
    • Evidence (submitted)
    • Uncertainties
    • FDA’s regulatory options ( adding contraindications, limitations of use, boxed warnings, etc.)
  •  FDA has also provided the benefit-risk framework as follows:
Dimensions Evidence and Uncertainties Conclusions and Reasons
Analysis of Condition
Current Treatment Options
Risk and Risk Management
Conclusions Regarding Benefit-Risk
  • It also focused on the role of patient experience data in in FDA’s Benefit-Risk Assessment and how to collect the same information in a structured manner.
  • The document has details regarding the structured benefit-risk planning during drug development and the details required for the interaction between the sponsor and the FDA for the benefit-risk assessment.
  • Finally, it also focused on the information regarding the additional analyses and the presentation of the risk-benefit consideration in marketing applications.

Industry Impact

The guidance provides valuable insights to the marketing authorization holder on the important key points in line with the FDA’s expectation that should be considered during the development of the benefit-risk assessment profile of a new drug and a biological product.


Benefit-risk assessment is a continuous process that doesn’t conclude with FDA approval. Post-market data sources, periodic reporting, and prompt communication of safety concerns all contribute to ongoing benefit-risk evaluations throughout a drug’s lifecycle. The FDA emphasizes the importance of communicating new information regarding safety concerns that could affect a drug’s benefit-risk profile. This guidance is a significant resource for the pharmaceutical industry, providing a comprehensive framework to assess the benefits and risks of new drugs and biological products.

This blog provides an overview of the FDA’s guidance, shedding light on its implications for the pharmaceutical industry. It emphasizes the critical role of benefit-risk assessment in drug development and post-market evaluation, underscoring the need for continuous monitoring and transparent communication with regulatory authorities.

Benefit-Risk Assessment for New Drug and Biological Products – FDA Guidance can be found here.

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