FDA Oversight of Unproven Drugs is Lacking: Government Report

A new congressional report from the Government Accountability Office (GAO) indicates that the 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.

The report, which was released this week, looks at a group of 90 drugs approved since 1992 through FDA’s accelerated approval process. The GAO found that although pharmaceutical companies often fail to provide FDA-required post-marketing data which shows that the drugs are working, the FDA has never removed any of the drugs from the market.

The accelerated approval process allows critical drugs designed to treat life-threatening or serious illnesses to go through an expedited approval process without years of testing and studies required by normal drugs. To obtain approval, drug makers must show that the drug is likely to work through surrogate endpoints, such as showing that a cancer drug shrinks tumors or an AIDS drug lowers the viral count.

In return for FDA’s expedited approval, the drug makers must provide post-marketing data that demonstrates the drugs are working as predicted. However, the GAO report found that 36% of drugs approved by the FDA under the process do not have the post-marketing data as required. In some cases, the drugs have been available for over a decade and generated hundreds of millions of dollars in revenue for the drug makers without such follow-up studies being completed.

The GAO report recommends that FDA clarify under what conditions it would withdraw one of these drugs off the market, most of which are used to treat cancer or HIV/AIDS. However, the report states that the FDA disagreed with GAO, saying that it did not need to clearly indicate when it would enforce its own rules to remove potentially dangerous or ineffective drugs approved under the accelerated approval process.

FDA officials have said that in many cases, the drugs approved under this process are life-saving drugs for which there are no replacements, and if the drugs are taken off the market, the patients will have no alternatives. However, the GAO report indicates that in some cases the drugs could be harmful to patients, and without the required post-marketing data the FDA may never know.

The GAO noted that the drug makers are under no pressure by the federal drug regulatory agency to provide the data or prove their drugs benefits actually pan out, since the FDA has not acted against a single drug approved under the accelerated approval process since its inception. The GAO found that not only has the FDA never enforced its accelerated approval process rules, but it has no rules or established procedures on how to do so.

The GAO report noted that enforcement of accelerated approval 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.”