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US: FDA finalizes guidance on real-world evidence in drug approvals

2023/09/01  US FDA

The 21st Century Cures Act (Cures Act), signed into law on December 13, 2016, is intended to accelerate medical product development and bring innovations faster and more efficiently to the patients who need them. Among other provisions, the Cures Act added section 505F to the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 355g). Pursuant to this section, FDA created a framework for a Real-World Evidence (RWE) Program to evaluate the potential use of RWE in regulatory decision-making for drugs.

FDA is issuing this guidance as part of its RWE Program to satisfy, in part, the mandate under section 505F of the FD&C Act to issue guidance about the use of RWE to help support approval of a new indication for a drug already approved under section 505(c) of the FD&C Act (21 U.S.C. 355(c)) or to help support postapproval study requirements.

For the purposes of this guidance, FDA defines real-world data (RWD) and RWE as follows:
• RWD are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.
• RWE is the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.

This guidance discusses the applicability of FDA’s investigational new drug application (IND) regulations under part 312 (21 CFR part 312) to various clinical study6 designs that utilize RWD. The guidance also clarifies the Agency’s expectations concerning clinical studies using RWD submitted to FDA in support of a regulatory decision regarding the effectiveness and safety of a drug (e.g., as part of a new drug application (NDA) or biologics license application (BLA)) when such studies are not subject to part 312. This guidance focuses primarily on clinical study designs that are non-interventional.

In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required.

For the purposes of this guidance, the term interventional study (also referred to as a clinical trial) is a study in which participants, either healthy volunteers or volunteers with the condition or disease being studied, are assigned to one or more interventions, according to a study protocol, to evaluate the effects of those interventions on subsequent health-related outcomes. One example of an interventional study is a traditional randomized controlled trial in which some participants are randomly assigned to receive a drug of interest (test article), whereas others receive an active comparator drug or placebo. Other examples of interventional study designs include randomized clinical trials with pragmatic elements (e.g., broad eligibility criteria, recruitment of participants in routine care settings) and single-arm trials.

For the purposes of this guidance, a non-interventional study (also referred to as an observational study) is a type of study in which patients received the marketed drug of interest during routine medical practice and are not assigned to an intervention according to a protocol. Examples of non-interventional study designs include, but are not limited to, (1) observational cohort studies, in which patients are identified as belonging to a study group according to the drug or drugs received or not received during routine medical practice, and subsequent biomedical or health outcomes are identified and (2) case-control studies, in which patients are identified as belonging to a study group based on having or not having a health-related biomedical or behavioral outcome, and antecedent treatments received are identified.

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