The intent of this guidance is to clarify for drug sponsors and other stakeholders how considerations about a drug’s benefits, risks, and risk management options factor into certain premarket and postmarket regulatory decisions that the Food and Drug Administration (FDA or Agency) makes about new drug applications (NDAs) submitted under section 505(b) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 355(b) as well as biologics
license applications (BLAs) submitted under section 351(a) of the Public Health Service Act (PHS Act).

This guidance first articulates important considerations that factor 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 to inform the benefit-risk assessment. It then discusses how sponsors can inform FDA’s benefitrisk 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. It also discusses opportunities for interaction between FDA and sponsors to discuss benefit-risk considerations in connection with the development of an NDA or BLA. This guidance concludes with additional considerations on benefit-risk assessments that inform regulatory decision-making in the postmarket setting.

This guidance pertains to benefit-risk assessments made to support certain regulatory decisions about NDAs or BLAs, from premarket approval through the postmarket setting. This includes decisions regarding any regulatory requirements for approval, such as inclusion of a boxed warning in approved labeling, postmarketing study requirements and commitments, and risk evaluation and mitigation strategies (REMS).

These regulatory decisions are made in accordance with specific, applicable legal and regulatory authorities and criteria.

This guidance touches on some of these authorities but does not attempt to list or address them all.

This guidance does not directly address other regulatory decisions that may occur throughout the drug development lifecycle, such as decisions regarding first-in-human trials of an investigational new drug and expanded access applications, which also may require FDA to consider information about the benefits and risks of an investigational or marketed drug for its proposed use. However, the concepts discussed in this guidance may also be relevant to these other types of decisions.