GAO: FDA fails to follow up on unproven drugs

WASHINGTON -- The Food and Drug Administration has allowed drugs for cancer and other diseases to stay on the market even when follow-up studies showed they didn't save lives, say congressional investigators.

And the agency has never pulled a drug off the market because of a lack of required follow-up about its actual benefits -- even when such information is more than a decade overdue, according to a report due out today from the Government Accountability Office.

When pressed about that policy, agency officials said they have no plans to get more aggressive.

The GAO found the FDA does little to track whether drugs approved based on preliminary results actually have lived up to their promise.

In 1992, the FDA began granting "accelerated approval" to novel drugs based on so-called surrogate endpoints, or laboratory measures that suggest the drug will make real improvements in patient health. HIV drugs, for example, are cleared based on their virus-lowering power, a predictor of survival.

Faster to market

Drugmakers favor the program because it allows them to get products to market sooner, without conducting long-term patient studies that can take years and cost hundreds of millions of dollars. However, a condition of the quicker approvals requires drugmakers to conduct follow-up studies to show the drug actually helped patients live longer.

But the GAO report, a copy of which was obtained by The Associated Press, identified several drugs still on the market that never lived up to their initial promise. And in the 16 years that the FDA has used accelerated approval, it has never once pulled a drug off the market, even when companies went more than a decade without submitting follow-up data.

"FDA has fallen far short of where it should be for patient safety," said Sen. Charles Grassley, R-Iowa, who requested the investigation.

Of the 144 studies the FDA has required under the program since 1992, more than one-third are still pending, according to the GAO. Investigators said the FDA does not regularly check whether companies are making progress on their required studies, although the agency is improving its oversight.

"According to FDA officials, this task was a lower priority compared to other responsibilities," the report says.

In the case of Shire Laboratories' low blood pressure treatment ProAmatine, the required study has gone incomplete for more than 13 years. The GAO found that ProAmatine has generated more than $257 million in sales, even though "the clinical benefit of the drug has never been established."

Shire did not respond to a request for comment Friday.

FDA's failure to act

In other cases, the FDA has failed to act even when company studies show drugs did not improve patient outcomes.

The FDA approved AstraZeneca's Iressa in 2003 based on early results showing it reduced the size of tumors. But later studies showed the drug did not help patients live any longer. The FDA has left the drug on the market, despite side effects including hundreds of reports of a sometimes fatal pneumonia.

AstraZeneca PLC said in a statement the drug is only sold through a limited access program and "is not available to new patients."

The GAO concluded that the FDA has no policy for pulling drugs off the market that were approved using surrogate endpoints. When GAO investigators confronted FDA officials about this lack of enforcement, they reportedly said it would be "difficult, if not impossible," to draft a standard policy for withdrawals, because drugs vary case-by-case.

Some consumers advocates say that's not good enough.

"The FDA has talked a lot about doing more enforcement, but this is an area where they're basically defending not enforcing the law," said Dr. Sidney Wolf, of the consumer advocacy group Public Citizen.

Wolfe said the lax policy sends a message to companies that there is no penalty for failing to complete studies.

The GAO recommends the FDA clarify when it will pull drugs off the market.

"As the scientific experts charged with overseeing the use of drugs it approves, FDA should be in a position to implement this recommendation," the report states.