WASHINGTON, D.C. – Today the Government Accountability Office (GAO) released its report investigating the Food and Drug Administration’s (FDA) oversight of and reliance on non-inferiority trials to establish the safety and effectiveness of new drugs. This report was produced in response to a request from Senator Grassley and Representatives Henry A. Waxman, John Dingell, Bart Stupak, and Edward J. Markey. The report examined characteristics of non-inferiority trials that FDA considered in making approval decisions for new drugs between 2002-2009. While the report described efforts within FDA to create a more rigorous review process, the report identified concerns regarding how the study design of non-inferiority trials may weaken the FDA’s ability to assess the safety and effectiveness of drugs.

The co-authors of the letter issued the following statements:

Senator Grassley said: “This study gives the FDA another reason to carefully consider its use of these sorts of trials when approving new drugs for the market.  Every step should be taken to strengthen drug reviews done by the FDA for the consumers who rely on and benefit from life-saving and life-enhancing pharmaceutical drugs.”

“Non-inferiority trials are an essential tool for evaluating the safety and effectiveness of certain kinds of critically important drugs such as antibiotics—but they pose difficult scientific issues,” said Chairman Waxman.  “I’m pleased to see that FDA’s March guidance clarified the standards for these trials.  I hope FDA will work with industry and academics to address remaining issues on how and when to best conduct these trials. Although the pipeline for antibiotics is critically bare, no one benefits from ineffective drugs—and so I encourage FDA and industry to continue to collaborate to do everything possible to ensure that safe and effective antibiotics are developed.”

Rep. Dingell said: “This GAO report provides a very good assessment of the concerns related to the use of non-inferiority trials and the recent strides FDA has taken to address those concerns.  Non-inferiority trials have a role in the drug review process.  However, they must always be designed and interpreted in a way that clearly demonstrates a high level of safety and efficacy.  I look forward to FDA’s continued progress in this regard.  American consumers should have no doubts about the effectiveness of the drugs they consume.”

Rep. Markey said: “The GAO report shows that these so-called ‘non-inferiority’ trials have often proved to be an inferior means of reviewing the safety and efficacy of new drugs. While the FDA has made strides to improve evaluations relying on non-inferiority trial data, the GAO report highlights important limitations of non-inferiority trials. I will continue working to investigate and implement empirically sound changes to ensure that the FDA can protect the public’s health and that the drugs in our medicine cabinets have gone through a thorough test of their safety and effectiveness.”

A link to the GAO report is available here: http://markey.house.gov/docs/gao._clinical_trails._report_august_2010.pdf

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