The FDA officially shifted from requiring two pivotal trials to one as the default for new drug and biologic approvals, announced in a New England Journal of Medicine article by Commissioner Marty Makary and CBER Director Vinay Prasad last week.145
This policy expands single-trial approvals to broader therapeutic areas like neuropsychiatry, metabolic, and immunology, previously limited to oncology and rare diseases, supported by confirmatory evidence such as biomarkers or real-world data.146
Experts describe it as an evolution rather than revolution, potentially shifting power to industry while maintaining high scrutiny on the single trial's quality, controls, and endpoints; FDA may still require two trials for common diseases or weak studies.12
The change aims to reduce development costs ($30m-$150m per trial), speed access to therapies, and align with advances in trial design, but raises concerns about safety, efficacy standards, and risk of approving borderline results.345
Implementation via guidance is expected in 3-6 months, applying to traditional, accelerated, and priority pathways.45
Sources:
1. https://www.biospace.com/fda/fdas-one-trial-policy-not-a-revolution-but-a-potentially-risky-evolution
2. https://www.jdsupra.com/legalnews/fda-issues-guidance-on-plausible-3477412/
3. https://mahometdaily.com/fdas-new-one-trial-standard-raises-questions-on-speed-vs-safety/
4. https://eyewire.news/news/fda-officially-shifts-to-one-trial-standard-for-new-drug-approvals
5. https://www.clinicaltrialsarena.com/news/fda-to-allow-one-pivotal-trial-instead-of-two-for-drug-approval/
6. https://www.truveta.com/blog/news/fda-trial-change-real-world-evidence/