Expanding the Use of Real-World Evidence Can Make FDA Regulatory Decisions Faster, Cheaper, and Just as Safe
- kenashman
- Jun 12
- 1 min read
Published 27 March 2025 Manhattan Institute
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.
Recent nominees for senior Department of Health and Human Services (HHS) leadership roles, such as Jim O’Neill, have proposed previously that the U.S. Food and Drug Administration (FDA) require only that new medicines are safe before they are sold, not that they are also effective.[1] In fact, O’Neill’s suggestion was the approach that FDA followed until 1962. Under this policy,[2] greater use of RWD and RWE could help facilitate researchers’ evaluation of a medicine’s efficacy after it is marketed to the public.
FDA reviews applications to market new pharmaceuticals and oversees clinical trials designed and sponsored by new drug developers. In these clinical settings, researchers conduct randomized controlled trials (RCTs) in which participants receive either an experimental drug or the standard of care. Then the researchers assess which group fares better. This rigorous process generates valuable information about a drug’s benefits and risks in an idealized setting, but it can be time-consuming and expensive. For treatments for rare diseases, this is especially challenging. The process for drug development and approval can benefit greatly by leveraging RWD, including routinely collected health information such as medical and insurance records. While not appropriate for all contexts, these data can supplement the information gleaned from traditional trials.
