The 21st Century Cures Act (Cures Act),2 18 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 program (RWE Program) to evaluate the potential use of realworld evidence (RWE) in regulatory decision-making.

 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 drug4  already approved under section 505(c) of the FD&C Act 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 39 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.

The contents of this document do not have the force and effect of law and are not meant to bind  the public in any way, unless specifically incorporated into a contract. This document is  intended only to provide clarity to the public regarding existing requirements under the law.

FDA guidance documents, including this guidance, should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in  FDA guidance means that something is suggested or recommended, but not required.


Considerations for the Use of Real-World Data and RealWorld Evidence to Support Regulatory Decision-Making for Drug and Biological Products