Why Should FDA Regulate Drugs?

by Food and Drug Administration (FDA)

Is the Food and Drug Administration in need of reform? Some critics of the agency, in Congress and outside, argue that the agency should be overhauled to make it work more efficiently. FDA supporters disagree, pointing to record-setting drug approvals and other agency accomplishments in recent years.

FDA's Center for Drug Evaluation and Research (CDER) is the part of the agency that regulates all drugs marketed in the United States: brand-name and generic, prescription and over-the-counter.

Since June 1994, the center has been directed by Janet Woodcock, M.D., a rheumatologist who worked in FDA's Center for Biologics Evaluation and Research for eight years before becoming CDER's director.

Despite her center's record 131 drug approvals in 1996, including new treatments for AIDS, Alzheimer's disease, and diabetes, and despite a much-reduced median review time of about 15 months in that year, some continue to question the agency's role in the drug review and approval process. And you, as a consumer, may wonder, "Why should a government agency even be involved in my decisions about the drugs I want to take?"

In the following interview, Woodcock gives her views on these issues.

Q. Why not trust consumers to decide for themselves which medicines work for them?

A. I don't think it's in the government's best interest to stand between people, especially those who are desperately ill, and their desire to take medicine. But that libertarian issue shouldn't be confused with the scientific issue of whether patients can tell what medicines work, because with almost any drug treatment we use today, they can't tell.

Doctors thought for years they could tell what worked. In the 1960s, for example, doctors were convinced that diethylstilbestrol, or DES, was terrific for preventing early miscarriages, and they gave it to thousands of women in pregnancy. "The women had miscarriages before, and I put them on this DES, and some of them didn't have miscarriages. So obviously, it's very effective," doctors thought.

In fact, when DES was actually subjected to scientific testing, it had no effect on miscarriage whatsoever. Not only was it absolutely ineffective, but unfortunately it had delayed negative health effects on the fetus.

We had a more recent experience like this with a heart rhythm drug. After people have heart attacks, they can have extra beats. And it's known that a percentage of people with those extra beats will have sudden death. Well, drugs were discovered that made the sudden beats go away, and people thought, "Wonderful! Make the beats go away, and sudden death will go away." The medicine became the standard of practice throughout the United States; everybody was using the drug.

There were some skeptics at the National Institutes of Health and FDA that said the drug ought to be tested. NIH set up a trial, and what they discovered shocked everyone: Yeah, the drugs make the beats go away, but the people who were put on the drugs had sudden death at a substantially higher rate than the people who were just left having the beats. The drugs actually made the problem worse, and maybe more probable.

Even the people who did the trial were later haunted by the fact that they had given some people that drug. They were people the researchers knew, and some of them died.

So the answer is, many, many very smart people have thought they knew what drugs would help them and what drugs would hurt them, and clinical tests again and again have proven them wrong. They didn't know.

Q. What is there to lose by giving people with life-threatening diseases like AIDS and terminal cancer access to whatever drugs they want?

A. If we didn't test drugs — if people could take whatever they wanted without any testing — there would be no way to tell whether any of the thousands, millions, of candidate drugs out there worked. So no one would ultimately benefit.

For people with life-threatening illnesses, even the patient groups don't agree on where the right balance is between identifying treatments that will really improve patients' health and allowing people to have immediate access to experiment with drugs that might work for them.

I think AIDS is a good example. We had a lot of discussions with the AIDS activists early on about access to treatments. And FDA put together many programs to allow people early access to those drugs even before they were approved.

But at the same time, companies did pursue testing to see if these agents worked. Ultimately, some drugs were dropped, because they didn't work or because they were so toxic that the risks outweighed the benefits. And good drugs were found and then approved by FDA.

Now we're decreasing mortality with HIV. So every person with HIV has a path they know they can take of drugs that will work to improve their health, and have been proven to do so. If we'd gone down the other path, and everyone had been able to try anything they wanted with no testing, then we'd still be at the same point so much later into the epidemic: Everyone would have total availability to all drugs, but we wouldn't know what worked.

Now some of the AIDS activists are actually telling us they want more rigorous testing because, as they study their disease and the treatments, they realize they need information to make choices about which drugs they should take, even among the approved drugs. They want FDA to mandate more big trials that would include combination therapy. "What if I start this combination early, versus if I take this single drug first? Which would help me to be in better health 10 years from now?" Those are the kinds of questions they want answered, and you can't answer those questions unless you do scientific testing.

Q. Isn't FDA infringing on drug marketers' freedom of speech when the agency restricts what is said in drug labeling and advertising?

A. There is a category of speech called commercial speech when you're making a sales pitch. So, while some other kinds of speech are less restricted, things that are promotional in nature may have certain constraints legitimately put on them by FDA.

For example, drug labeling and advertising must be balanced and not misleading. In my opinion, consumers want truthful information; they want a lot of information, not hype.

Because people would like to receive all the latest information on a drug from the manufacturer, there has been a lot of debate about uses that are considered "off-label" — not approved by FDA.

Obviously, medical science doesn't happen in spurts, but continuously. After a drug is out on the market, health professionals continuously experiment with new uses. FDA thinks that's appropriate and doesn't want to restrict that kind of use of drugs. But we don't currently permit manufacturers to promote these new uses until it's proven that they work and are safe.

To help the situation, we've put out a draft guidance on how much information a manufacturer needs to get a new use put on the label. We think that will help to some extent.

Last year, the center approved 118 new uses for drugs that were already approved, way up from a few years ago. We think that manufacturers are motivated to send in applications for new uses because they know that the agency has been approving them promptly if they work.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.