FDA’s Approval Process Faces Challenge in New Senate Bill

This time last year, efforts to overhaul the federal Food and Drug Administration’s regulatory procedures had reached a level of high passion. Republican lawmakers were calling Commissioner David A. Kessler nasty names and vowing to block his attempts to regulate tobacco. Newspapers and the airwaves were saturated with ads condemning the agency for “killing” Americans by taking too long to approve new drugs and medical devices.

Nevertheless, a bill aimed at changing the way the FDA operates never got past legislative first base when critics argued that it would weaken the agency’s ability to protect the public from dangerous products.

This year, with Kessler having resigned to become dean of Yale University Medical School and the tobacco issue defused by a proposed settlement between states and cigarette makers, the temperature for reform seems to have fallen sharply.

Still, a bill similar to last year’s proposal has made it to first base again--sent to the floor by a key Senate committee. And critics worry that, outside the spotlight, the measure might make it home this time.

“I agree that anger toward Dr. Kessler was behind some of the energy last year, but the bill still has a certain amount of momentum,” said one senior FDA official who requested anonymity.

Despite the cooler climate this year, the basic issues haven’t changed. In fact, the debate that swirls around the agency’s mission is much the same as it has been for decades: finding the proper balance between protecting and overburdening the public.

On the one side are lawmakers and officials of the drug and medical-device industry who seek to “streamline” the agency, mainly by having it accelerate the approval process for experimental drugs and devices.

On the other side are consumer groups who believe that such a speedup will weaken the FDA, which oversees products accounting for 25 cents of every consumer dollar spent. They argue that it would leave the public vulnerable to potentially dangerous medicines and merchandise.

The Senate Labor and Human Resources Committee recently approved the FDA bill; it awaits action by the full Senate, which could come as soon as this week. Sen. James M. Jeffords (R-Vt.), the committee chairman, calls it “a moderate proposal” that will ensure “a strong FDA.”

The drug and medical-device industries back it, contending that the changes will make the agency more efficient without compromising safety. “It undermines consumer protection not one bit,” said James S. Benson, a former FDA official who is now executive vice president of the Health Industry Manufacturers Assn.

But Joan Claybrook, president of the consumer group Public Citizen, describes the bill as “a special-interest grab bag for the pharmaceutical and medical-device industry.”

The measure contains many of the same elements as last year’s proposal, including provisions to reduce the number of new drug studies required to gain FDA approval as well as to let outside reviewers evaluate experimental devices.

The proposal on experimental devices is sparking significant debate. FDA officials have acknowledged that they could do a better job when it comes to medical devices--which includes everything from heart valves to bandages--but there is disagreement on whether this bill is the way to solve the problem.

The legislation would allow experimental devices to be scrutinized by private experts hired by the industry, which would also foot the bill. The FDA would retain oversight over the reviewers by certifying them in advance and passing final judgment on the devices.

Supporters maintain that this would hasten device approval. Opponents fear it would raise serious conflict-of-interest questions.

FDA officials, who are evaluating a pilot program that uses third-party reviewers, say such a program might work if it didn’t become too big to manage. They also oppose the idea of removing from the FDA the review of critical medical devices, such as those that are implanted in the body.

Another controversial provision is expected to be added to the bill as an amendment during floor debate. The proposal would allow drug companies to let doctors know the results of studies of so-called off-label uses of licensed drugs, even if the studies were quite small.

The term “off-label” refers to the practice of prescribing a drug for uses or among patient populations that have not been approved by the FDA.

Under current FDA statutes, drug manufacturers cannot distribute such information, which typically is published in medical and scientific journals; it is considered illegal promotion. Physicians, though free to prescribe approved drugs for off-label purposes, generally are reluctant to do so until large-scale studies have been conducted and the FDA has signed off on the new use.

Many patient-advocacy groups seek greater access to drugs for off-label purposes and say the amendment would help achieve that aim. FDA officials, however, are nervous about this concept. “Doctors appropriately rely on journal articles all the time, but there are two problems associated with [relying on small studies]: Companies lose the incentive to do the [larger] studies and, occasionally, you’ll have a medical disaster,” said one agency official who requested anonymity.

The recently revealed heart-valve problems associated with the use of the popular weight-loss drug combination known as fen-phen is one example of off-label prescribing that caused a potentially serious situation.

The two-drug combination was not approved by the FDA; the drugs are licensed only for individual short-term use. But prescriptions soared after one small study showed that the two drugs--fenfluramine and phentermine--worked better for weight loss and seemingly had fewer side effects when used together.

Doctors, however, now have identified a potentially serious heart-valve abnormality among some women taking the drug combination. As of Monday, the FDA was reviewing 49 such cases.

The fen-phen case could undercut support for an amendment to liberalize the availability of off-label studies. But the proposal may have received an unexpected boost by a survey released Monday that found cancer doctors overwhelmingly discount FDA-approved label information and instead use studies about off-label use when prescribing drugs.

The survey, conducted by the American Enterprise Institute, a think tank, reported that 85% of the doctors said FDA labels have “little influence” or “practically no influence at all” in their treatment of patients. Also, 75% of the doctors said FDA rules about off-label information hinder doctors’ efforts in getting current information on cancer treatments.

As part of the bid to pass the FDA bill, supporters included in it a provision to extend the life of a popular existing program known as the Prescription Drug User-Fee Act, which is due to expire Sept. 30.

Under the law, companies must pay the FDA a fee for each drug submitted for review. The money is used to hire additional agency drug reviewers.

Most agree the program has accelerated drug reviews. But some, particularly those opposing other parts of the bill, would prefer that PDUFA, as it is known, be acted upon separately. The bill’s sponsors admit that they deliberately included it as an incentive for passing the rest of the bill.

Times staff writer Heather Knight contributed to this story.