FDA Drug Approvals Have Variable Clinical Trial Evidence Record

MedicalResearch.com Interview with:
Nicholas S. Downing, AB
Yale University School of Medicine
New Haven, Connecticut

MedicalResearch.com: What are the main findings of the study?

Answer: In our systematic review of all new drugs approved by the FDA over an 8 year period, we found that there was real variability in the quality and quantity of clinical trial evidence used as the basis of the agency’s approval decisions. Some drugs were studied in multiple randomized, double-blinded, controlled clinical trials that provide very helpful information for patients and physicians. However, other drugs were studied in clinical trials that did not produce as much information about their safety and effectiveness.

MedicalResearch.com:  Were any of the findings unexpected?

Answer: Several features of this variability were notable.

First, we found that just over one third of drugs were approved on the basis of a single pivotal trial, rather than the traditional standard of two.
Second, almost half of drugs were approved the basis of surrogate outcomes.
Finally, only 40% of drug approvals involved a trial comparing the new drug to existing therapy; such information is important because it can help patients and physicians determine the most appropriate indication for its use.

MedicalResearch.com: What should clinicians and patients take away from your report?

Answer: Patients and physicians should be aware that not all FDA approvals are created equally. Some are approved on the basis of strong clinical trial evidence, while others on more preliminary evidence.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Answer: It is important that all drugs, in particular those approved on the basis of preliminary evidence, continue to be studied in the post-marketing period. Future research should evaluate whether this information is being generated in a consistent manner.

Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012

Downing NS, Aminawung JA, Shah ND, Krumholz HM, Ross JS. Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012. JAMA. 2014;311(4):368-377. doi:10.1001/jama.2013.282034.


Last Updated on April 3, 2015 by Marie Benz MD FAAD