GAO Report Calls Out FDA's Poor Oversight of Accelerated Drug Approvals

October 29, 2009
Angie Drakulich

Angie Drakulich was editorial director of Pharmaceutical Technology.

ePT--the Electronic Newsletter of Pharmaceutical Technology

The US Food and Drug Administration needs to enhance its oversight of drugs approved on the basis of surrogate endpoints, according to a September report from the Government Accountability Office (GAO) that was released this week.

The US Food and Drug Administration needs to enhance its oversight of drugs approved on the basis of surrogate endpoints, according to a September report from the Government Accountability Office (GAO) that was released this week.

FDA established its accelerated approval process in 1992 to expedite drug approvals for products that treat serious or life-threatening illnesses. The agency approved 90 such applications between 1992 and Nov. 20, 2008. These approvals are based on the drug’s impact on a surrogate endpoint such as blood pressure rather than on direct clinical evidence such as the drug’s ability to prevent a stroke, according to the GAO report. The agency then relies on postmarketing studies to further assess the drug’s effectiveness, which is where problems seem to come into play, according to the GAO report.

Specifially, GAO found that the agency’s ability to effectively monitor postmarketing studies is weak. Furthermore, the agency does not have clear procedures for using its authority to withdraw a drug from the market if a manufacturer fails to complete a postmarketing study or if such a study fails to confirm a drug’s clinical benefit. The GAO report states that FDA has never exercised its authority to withdraw a drug in such conditions even if a required postmarketing study, in one instance, was still not completed after 13 years.

GAO recommends that FDA clarify the conditions under which it would expedite the withdrawal of drugs under its accelerated approval process as a way to enhance the agency’s oversight.

FDA disagreed with the need for such clarification in its drug-withdrawal procedures, according to the GAO report. Although the agency does have a plan to strengthen its oversight, “the agency has taken a passive approach to enforcing confirmatory study requirements,” says GAO.