12 Jun Drugs Receiving Accelerated FDA Approval Often Have Flaws In Their Evidence Base
MedicalResearch.com Interview with:
Dr. Naci
Huseyin Naci, PhD
Assistant Professor of Health Policy
LSE Health
Department of Social Policy
London School of Economics and Political Science
London, United Kingdom
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: An increasing proportion of novel therapeutic agents are entering the market on the basis of expedited development and approval programs. The Food and Drug Administration’s (FDA) Accelerated Approval pathway is one such expedited approval program. In our study, we examined the dynamics of research on drugs receiving accelerated approvals. We were particularly interested in the timing and characteristics of research studies including drugs with accelerated approvals.
Our primary findings are the following:
- First, there is an abundance of research on drugs receiving accelerated approvals. Yet, the majority of this research (about 70%) is of poor quality. Ideally, these drugs are evaluated in so-called randomised controlled trials to establish their efficacy and safety. However, only about a third of all existing studies are randomised controlled trials.
- Second, a substantial share (about 30%) of the existing research on these drugs is in areas not approved by the FDA. This may be indicative of industry research practices in trying to prioritise identification of new uses for drugs receiving accelerated approvals instead of strengthening their evidence base.
- Third, when focusing on well-designed studies, only about a half actually evaluate the effectiveness of the accelerated approval drugs. The rest appears to use the accelerated approval drug as background therapy. Interestingly, these two types of studies are conducted concurrently. In other words, while one research group is trying to find out if an accelerated approval drug is effective, other research groups are already using it as part of a background regimen when testing the effectiveness of another, potentially newer drug
MedicalResearch.com: What should readers take away from your report?
Response: Drugs receiving accelerated approvals often have major flaws in their evidence base. Research practices on these drugs rarely meet the information needs of patients, doctors, and other decision makers in health care systems.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: Further research is needed to examine the nature of the thousands of small non-randomised studies conducted on drugs receiving accelerated approvals.
MedicalResearch.com: Is there anything else you would like to add?
Response: We have no conflicts of interest to report. The article was published in The Milbank Quarterly. http://onlinelibrary.wiley.com/doi/10.1111/1468-0009.12261/abstract
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Last Updated on June 12, 2017 by Marie Benz MD FAAD