FDA’s Fast Track for Medications Called ‘Broken’

The government’s fast-track system for making new medications quickly available to treat the deadliest illnesses has become a route for companies to market drugs without fully proving their effectiveness or safety -- either before or after they go on the market, according to a congressional report to be released today.

The report found that pharmaceutical companies receiving accelerated approval from the Food and Drug Administration often fail to follow through on promises to do complete field trials and other studies after their drugs reach the market.

The system, created largely in response to the AIDS epidemic, appears to be “broken and failing to ensure patient safety,” said the report by the staff of Rep. Edward J. Markey (D-Mass.).

An industry trade group, however, said there was no evidence that drug recalls had increased as a result of the accelerated approval process.

Under the system, drug makers can get the go-ahead -- after an abbreviated series of clinical trials -- to market medications for life-threatening illnesses. As a condition of approval, the FDA requires the companies to complete fuller studies, as they must do for other medications.

But drug firms have completed about half of the post-market clinical studies that they pledged to conduct for accelerated approval medications, the report found. Of 91 studies committed to since the process was instituted in 1992, 42 have not been completed. Of those, half of the promised studies -- 21 -- have not been started, the report said.

Fast-tracked medications can be sold without special restrictions or warnings. Some patients may not be aware that the drug they are taking is still under study.

“The American people have been left in the dark about the contingent nature of the approval of these drugs,” Markey said. “Patients are paying to be human guinea pigs in real-world trials that have no end.”

As a senior member of the Energy and Commerce Committee, which oversees the pharmaceutical industry, Markey said he would introduce legislation to impose penalties on companies that failed to conduct such studies with “due diligence.” The bill also would also require companies to inform patients and doctors that accelerated approval of a drug was conditional.

An FDA official said the agency could not comment on the report because it had not received a copy. “Accelerated approval allows the agency to give earlier access to promising new therapies for patients with serious and life-threatening conditions,” FDA spokeswoman Suzanne Trevino said.

Caroline Lowe, vice president of scientific and regulatory affairs for the Pharmaceutical Research and Manufacturers of America, defended the system, noting that the recall rate of 3% for new medications had remained stable since the inception of accelerated approval.

“It can reasonably be concluded that a system such as accelerated approval has not resulted in the introduction of unsafe products to the marketplace,” Lowe said.

However, an accelerated approval drug to treat multiple sclerosis gained notoriety this year when it was pulled off the market after a small number of patients developed a rare, often deadly infection seen in AIDS patients. The drug, Tysabri, had been approved last fall, based on results from the first year of two clinical trials designed to last two years. A patient in one of the studies was diagnosed with the infection in February. At least one patient died.

In Tysabri’s case, the manufacturer did follow through with the promised studies.

One academic authority on the accelerated approval process said the FDA should set deadlines for all such studies. If a company fails to comply in time, its drug should be deemed experimental.

“When the [maker] of a drug is in a pre-market setting, there is a sense of urgency to do a reliable assessment,” said Thomas Fleming, chairman of the biostatistics department at the University of Washington. “When accelerated approval has been provided, there is a loss of urgency.”

Fleming, who also has served on FDA advisory committees, published a study this year calling on the agency to overhaul its system.

“While accelerated approval was intended to provide access to therapies in life-threatening situations where there was an unmet need, it has not been carried out in a way that meets the true intention,” he said Tuesday.

“There are way too many instances where the interval between granting accelerated approval and having the completed clinical trials is unacceptably long.”