Expanding the Use of Real-World Evidence Can Make FDA Regulatory Decisions Faster, Cheaper, and Just as Safe
- kenashman
- May 8
- 1 min read
By Randall Lutter 27 March 2025
New information technology gives the U.S. an opportunity to accelerate the development, review, and approval of new medicines. This can be done through greater use of real-world data (RWD) and real-world evidence (RWE) rather than through the results of expensive clinical trials alone.
The 2016 21st Century Cures Act[3] directed FDA to facilitate the use of RWD and RWE in the review of drug marketing applications and labeling changes—yet progress has been glacial, despite the potentially dramatic cost reductions. While FDA has issued numerous industry-facing guidance documents on RWD and RWE, the agency has not reported the challenges to increasing the use of these data and evidence and has not adopted quantitative goals for such use.
FDA should increase the use of RWE in drug regulation by increasing transparency into its use of RWE, supporting more research into the reliability of RWE, and committing to specific quantitative goals. Specifically, one-half of all supplemental applications for new uses of drugs should incorporate RWE by 2030. Also, there should be increased use of RWE in novel rare disease treatments. If FDA fails to adopt these goals, Congress should require them. In addition, an independent organization such as the National Academies of Sciences, Engineering, and Medicine (NASEM) should evaluate what regulatory obstacles in the U.S. hinder the use of pragmatic trials and should make recommendations for legislative or regulatory actions that might help overcome such obstacles.
