The US Food and Drug Administration (FDA) published its final guidance for industry on the use of real-world data (RWD) and real-world evidence (RWE) in supporting regulatory decisions for drugs and biologics on August 30, 2023.1 This guidance document represents the culmination of the draft guidance with the same title that was initially issued on December 9, 2021. According to the FDA, the guidance applies to any RWD, from registries to medical claims, and includes data on products used in clinical practice under an emergency use authorization.
This guidance represents the FDA’s current viewpoint and offers non-binding recommendations for RWD access, collection and analysis transparency, study monitoring and reporting, and the sponsor’s responsibilities. The guidance mainly centers on non-interventional clinical study designs, such as observational cohort studies and case-control studies. It also describes the FDA’s perspective on potential applications of RWD in interventional studies conducted under an investigational new drug (IND) application. For example, RWD might help identify potential participants for a randomized controlled trial, choose study endpoints, or serve as a comparison group in an externally controlled trial.
Figure 1: Topics included in the FDA’s Final Guidance on RWE/RWD.
This section of the guidance discusses the applicability of the US Code of Federal Regulations, Title 21, Part 312 (Investigational New Drug Application) to studies involving the use of RWD. Part 312 outlines the procedures and conditions governing the use of investigational new drugs, including the requirements for an IND submission to the FDA and its review.
The FDA’s final guidance also lays out considerations for RWD in non-interventional studies, such as observational studies or trials in which treatments are prescribed as originally authorized.
The FDA guidance offers valuable pointers for using RWE in regulatory decisions. By following this guidance and collaborating with the FDA from the inception of a study, industry professionals can better use RWE to improve drug and biological product safety and effectiveness evaluations.
Axtria’s solutions in RWE and HEOR take an integrated approach, encompassing data infrastructure, analytics and talents, processes, and governance for agile and actionable insight deliveries. Axtria offers integrated evidence generation by leveraging a full spectrum of analytics (descriptive, diagnostic, predictive, and counterfactual) across the product lifecycle, encompassing pre-launch, launch, and post-launch. Our innovative solutions bridge the evidence gap between clinical trials and real-world practice. We can help clients in navigating the ever-evolving regulatory landscape.
We hope you find this summary of the FDA’s final RWE guidance valuable. If you would like more information on Axtria’s solutions in RWE and HEOR, please contact us at connect@axtria.com.