William S. Merrell Co. of Cincinnati first applied for U.S. approval of Thalidomide in September 1960. Last week the Food and Drug Administration approved it. New Jersey-based Celgene Corp. may now market Thalidomide, under strict controls, to treat a rare condition associated with Hansen's disease (leprosy). The agency's initial rejection of Thalidomide "sealed the agency's reputation as the finest consumer safety authority in the world," the New York Times reported recently. Now, almost four decades later, the unsealing begins.
Or continues, really. Since 1960 the FDA has very quietly contrived to make Thalidomide available for "compassionate use" and "experimental programs." And for years the FDA has quietly tolerated clubs that bought Thalidomide in Brazil for underground distribution in the U.S. The agency moved to shut down the illegal market only when it was at last ready to approve a legal one.
Defects Avoided
On one side of its Thalidomide ledger, the FDA can record thousands of birth defects avoided when a junior official at the agency took her time reviewing Merrell's original application. Delay was all she contributed, but that was good enough. For while she hesitated, a German scientist identified Thalidomide's dreadful power to halt limb development in the early stages of pregnancy.
There is, however, much tragedy on the other side of the Thalidomide ledger, too. Rapidly dividing cells create a fetus's arm; they also create cancerous tumors. It is thought that Thalidomide injures fetuses by halting the proliferation of blood vessels, an effect that also shows enormous promise for starving malignant tumors. Scientists have known for years that Thalidomide holds great promise in the treatment of macular degeneration in the eye, and in the treatment of brain and of breast cancer. Thalidomide also powerfully affects the immune system; the "buyer's clubs" the FDA unofficially tolerated were organized by AIDS patients.
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All strong medicines are strong poisons too. And just as disease is viciously selective and personal, so too are many of its treatments. This principle is highly inconvenient for an agency charged with monitoring the pharmacopeia of an entire nation from the banks of the Potomac. The FDA has now made official pronouncements about only two of Thalidomide's effects--on healthy pregnant women, and on victims of Hansen's disease. There are hundreds of other effects.
What the FDA finally did last week was politically deft. It approved Thalidomide--but only to treat one very rare disease, of which there are some 50 cases a year nationwide. Celgene may not lawfully publicize other possible uses.
But doctors are now technically free to prescribe Thalidomide as they see fit. Unofficially, the FDA knows full well there will be thousands of such "off-label" prescriptions. So the agency silences the thousands, while taking official responsibility for only the 50. If Thalidomide ever turns out to be modestly effective in treating something common like breast cancer, that effect won't make it on to the label for years, if ever. The risk of new Thalidomide babies must inevitably increase in proportion to the frequency of approved use, particularly approved use by women. It will be politically very hard for the FDA ever to let that risk grow large, notwithstanding lifesaving benefits on the other side of the ledger.
Even so, new Thalidomide babies will assuredly be born. Year by year, the FDA will be pressed to reinstate a complete ban, even as it will be pressed to expand the list of approved uses. Time and again, the agency will have to rebalance the fate of unborn children against that of mothers with breast cancer, fathers with AIDS, or grandparents who are going blind. And while the agency reckons up just how to do that, sick people will wait.
It is said that the FDA cannot--should not--move any faster than the progress of verified medical science. But in fact it moves a lot slower. Placing a layer of bureaucratic review on top of all the normal processes of medical science can have no other effect. The FDA can indeed force medical scientists to conduct more trials, or investigate more side effects. But more research is unconditionally good only when it uncovers things that are unconditionally bad. The FDA can indulge the presumption that everything it doesn't know should be shunned. AIDS patients cannot. The FDA can set its own, leisurely timetable. Cancer patients cannot.
Does federal oversight at least impose the norms of rigorous science on an industry that would not abide by them otherwise? The post-Thalidomide FDA legislation of the 1960s "ushered in a whole new era of scientific evaluation of medical therapies," declares David Kessler, a former FDA commissioner. "It's why today we know whether a drug works." What nonsense. Scientists established the efficacy of vaccines, antiseptics and antibiotics long before lawyers arrived to supervise their work. Medical science does not require Washington's rules or approvals to "know whether a drug works." The suggestion that political process is what makes science reliable is one worthy of Lysenko and the master he served. Real scientists stopped saying such things around the time of Galileo.
The truth is quite the opposite: Superposing a political layer of review on scientific research has a corrosive effect on the science itself--at least as corrosive as the pursuit of profit in the private sector. No private company prospers for long selling products that kill, maim or injure, least of all in an era when trial lawyers can bankrupt a company on the strength of even pseudoscientific phantasms. Washington agencies, by contrast, can and quite often do trim their science to fit the political winds. Statistics sufficient to persuade Washington that secondhand smoke injures children, or that smog increases newborn mortality rates, are rejected out of hand if they show that abortion increases the risk of breast cancer.
Customized Therapies
Most ordinary, healthy people probably still take some comfort in the thought that a diligent, generally competent, well-meaning federal agency is keeping an eye on the contents of their medicine cabinets. But we live in an age of enormously rapid progress in medical science. Impelled by genetic science, we are progressing toward ever more individualized, customized therapies. Some therapies already depend on extracting, modifying and cultivating cells, tissues or organs from the patient's own body, or from close relatives. General-issue tailoring of your medicines is fine if you happen to stand smack in the statistical middle of everything, but few real people do. And in the direst circumstances, the best therapies will often be the ones on the edges of science, well outside the bounds of the truths that have been fully certified in Washington.
Not long ago, the FDA approved Accutane, a powerful antiacne drug and a strong teratogen too. The warnings are all there, but babies with severe deformities linked to Accutane have been born nonetheless. The same will happen with Thalidomide: No level of warning will prevent it. As the father of three young children, I can hardly bear even to write down a truth so terrible; I cannot imagine what it's like to confront it personally. But children and adults with cancer, or AIDS, or failing eyesight, or Hansen's disease, confront terrible truths, too. I am glad it is not my responsibility to trade one Thalidomide child's welfare against another's, as the FDA is empowered and required to do. I am inclined to believe that it should not be the FDA's final power or responsibility, either.
Mr. Huber is a fellow of the Manhattan Institute.