Before 1962, the FDA regulated for drug safety, but not for drug efficacy. If the FDA returned to regulating only for safety, that would imply that Phase 3 randomized clinical trials would no longer be mandated. Phase 3 trials are usually more expensive than the Phase 1 and Phase 2 trials combined. They cost a lot more, and usually take a lot longer. If the FDA no longer mandate Phase 3 trials we will have more drug innovation, more quickly, and have much lower costs. And we will have more freedom.
(p. A13) From 1938 through 1962, the Food and Drug Administration required proof of safety before drug approval but not proof of efficacy. The approach was abandoned due to a significant misunderstanding of the thalidomide tragedy—when thousands of babies outside the U.S. were born with severe birth defects.
The issue with thalidomide was a failure of safety, not efficacy. But under pressure to react, Congress required, through the Kefauver-Harris Amendments of 1962, proof of efficacy before granting marketing approval. The new rule addressed a problem that didn’t exist and, in doing so, imposed a substantial new cost burden.
Before 1962, developing a drug took about two years. Now it takes 12 to 14 years. Since 1975 real development costs have risen about 7.5% a year, roughly doubling every decade. Today, we estimate that bringing one successful drug to market costs about $9 billion on average.
For the full commentary, see:
(Note: the online version of the commentary has the date Oct. 17, 2025, and has the same title as the print version.)