FDA Would Like Sponsors to Modernize Clinical Trials
​[Thursday, March 21, 2019]

FDA prefers that legacy clinical trial programs where a given product is tested through long clinical trials with generalized designs be replaced with modernized clinical development programs containing enrichment strategies, adaptive designs, master protocols, and operations supported by mobile technologies. Last week, FDA finalized two older guidance documents, one on enrichments strategies describing ways to identify high-risk or highly responsive patients for clinical trials to increase the probability of successful trial outcome, and another on risk-based monitoring to increase trial efficiency and prioritize resources. The draft of both these Guidance Documents had been released 5-6 years ago but there has been unsatisfactory adaptation of these trial modernization tools by the industry. In its announcement for these new Guidance Documents, FDA emphasized the need for innovative trial designs and that it will support creative development plans to facilitate faster to market strategies for product development. There is more than a decade of experience demonstrating FDA’s acceptance of programs that cut unnecessary clinical and non-clinical testing prior to market approval. Recently there also has been a push to increase the use of real world evidence not only for post-market development but also in lieu of pre-approval clinical trials, with emphasis on reduction of testing and faster approval. In additional, for exceptional products, using the breakthrough therapy designation pathway, FDA now has pathways to approve products based on fewer clinical trials, some even with one trial and in a few cases, without any additional trials. The onus of responsibility now lies with developers to take advantage of the new regulatory paradigms to reduce cost for development and faster access to patients. Modernized clinical programs are no longer available only for special cases but should be used for all products.