NOVEMBER 2009
FDA emphasizes overall success of program in providing treatment to patients with life-threatening illness
The U.S. Food and Drug Administration (FDA) has failed to follow up on unproven drugs that are put on the market through an accelerated approval process, and has no mechanism in place to remove those drugs when they prove to be unsuccessful or dangerous. This was the finding of a report recently released by the Government Accountability Office (GAO), the investigative arm of Congress, after it examined 90 drugs approved through the accelerated process from 1992 through 2008.
FDA established the accelerated approval process in 1992 to expedite approval of therapies that treat cancer and other serious or life-threatening illnesses. Pharmaceutical companies may bypass years of testing and studies required for approval by less essential drugs if they can show that their product is likely to have a positive impact on a surrogate endpoint, such as shrinking a cancer tumor, instead of more direct clinical evidence, like survival. In exchange, drug sponsors are expected to conduct research and provide the FDA with post-marketing data that demonstrates that the drugs are working.
GAO launched its investigation after concerns were raised by members of Congress about the regulatory agency's reliance on surrogate endpoints and its oversight of post-marketing studies. It found that 36 percent of drugs approved by the FDA under the accelerated process have not been followed up on with post-marketing data by pharmaceutical companies and the FDA has not acted against a single drug approved under the accelerated approval process since its inception.
The GAO report noted that enforcement of post-marketing requirements does not appear to be a priority for the FDA. "Although FDA has authority to expedite the withdrawal of a drug from the market if a sponsor does not complete a required confirmatory study with due diligence, or if a study fails to confirm a drug's clinical benefit, it has not specified the conditions that would prompt it to do so," the report states. "It has never exercised its authority, even when such study requirements have gone unfulfilled for nearly 13 years."
The GAO report concluded with the recommendation that FDA clarify under what conditions it would withdraw a drug from the market. FDA acknowledged that, in general, oversight of post-marketing studies has been inadequate and should be improved. However, it disagreed with GAO's recommendation, asserting that it would be "difficult, if not impossible," to draft a standard policy for withdrawals given the unique circumstances of individual drugs. FDA emphasized the success of the accelerated approval program, which it said has "provided millions of patients access to new treatments sooner than would have been possible under the traditional approval process."
Senator Charles Grassley (R-Iowa), who requested the investigation, issued a statement following the release of the report expressing his hope that it "should serve as an impetus for the FDA to improve the post-market surveillance of these drugs, giving patients and their doctors meaningful information and necessary safeguards."
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Read more from the November 2009 Edition of the AACR Cancer Policy Monitor:
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