GAO says US FDA needs to enhance oversight of approved drugs

A report issued October 26 by the USA's Government Accountability Office (GAO) has criticized the Food and Drug Administration for not following up on studies for some drugs that get approved after data suggest they are effective on a marker that stands in for the course of disease.

This accelerated approval process, established for drugs treating serious illnesses like cancer and HIV/AIDS, is supposed to move medicines along that could save or extend patients' lives. The problem is that these drugs are then supposed to go through "post-marketing studies" to confirm the drugs are safe and effective, so they can stay on the market. The GAO report also contends that the FDA has never pulled a drug off the market due to a lack of required follow-up about its actual benefits, even when such information is more than a decade overdue.

The GAO recommends that the FDA clarify the conditions under which it would utilize its authority to expedite the withdrawal of drugs under its accelerated approval process. However, the report noted that the FDA disagreed with the need to develop such clarifying guidance. The GAO believes doing so would enhance FDA's oversight.

FDA explains difficulties

The FDA responded that the report paints an overly negative picture of its so-called "accelerated approval" program, which is only used to approve drugs for the most serious diseases. In response, FDA officials explained that, given the unique circumstances of individual drugs, it would be "difficult, if not impossible" to draft a standard policy for withdrawals. The regulator, which indicated it has taken steps to improve its oversight, said that withdrawing a product would sometimes mean eliminating the only available treatment for a condition.

"Millions of patients with serious or life-threatening illnesses have had earlier access to new safe and effective treatments," thanks to the program, the FDA said in its response to the report, quoted by The Associated Press.

Since 1992, the FDA has approved 90 applications for drugs this way, bypassing the normal process. But the GAO says post-marketing studies were only completed for two-thirds of the quickly approved drugs, and none were pulled from the market.

Defending its approach, the FDA says that a blanket process for pulling drugs off the market would not work, pointing out that two drugs mentioned in the report that never finished post-marketing studies are the only medications on the market for the condition they treat, or they only show positive effects in certain patients, even if they don't work for everyone.

According to the GAO, the FDA has not routinely been reviewing sponsors' annual submissions on the status of studies in a timely manner. It has little in the way of readily accessible, comprehensive data to monitor studies' progression and does not consider such oversight a priority. It admits that the FDA is implementing initiatives to improve its oversight, but it is too early to tell if they will be effective.

Although the 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. It has never exercised its authority, even when such study requirements have gone unfulfilled for nearly 13 years, the GAO states.