Real-World Evidence: Agency is ready for discussion
Expenses related to the conduct of large traditional clinical trials have been growing steadily for years. When clinical trial results are applied to the clinical setting, real effectiveness and safety of a new drug turn out to be different from data obtained in clinical trials previously. That’s why both researchers and industry pay very close attention to real-world evidence.
Recently FDA employees published an article on perspectives of using real-world evidence to confirm effectiveness and safety of a new drug, in The New England Journal of Medicine. Authors warn that such information cannot be used easily in drug development process. Furthermore, the term “real-world evidence” is hard to define because such evidence is very heterogeneous and hardly classifiable.
FDA staff underlines that terms “randomization” and “real-world evidence” are not confronting each other. For instance, randomization procedure is used in ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-Term Effectiveness) trial despite the fact it is a real-world study. Thus, a real-world study should not necessarily be non-interventional.
Information gathered in clinical setting counts for a large proportion of effectiveness and safety data in orphan drug development process. FDA is going to meet with patients, healthcare providers, and sponsors to find the best way of regulation in real-world evidence area. Based on this information, draft guidance on how such evidence can be applied to assess safety and effectiveness in both premarketing and postmarketing regulatory requirements will be published.