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On July 16, the Food and Drug Administration granted marketing approval for a powerful pharmaceutical to treat Hansens disease (better known as leprosy). Although the drug was approved only for treating leprosy, its availability means that physicians can prescribe it for any illness they feel appropriate. Thats good news, because this particular drug holds promise for the treatment of a raft of other conditions, including several cancers, lupus, tuberculosis, and macular eye degeneration. The drug is not new, however. It is more than forty years old. Its called thalidomide.
Thalidomide was introduced in a number of European countries during the 1950s, as a sleeping aid and a treatment for nausea. Tragically, more than 10,000 European children were born with severe birth defects after their mothers took it during pregnancy for morning sickness.
Laws then in effect in the United States, requiring manufacturers to demonstrate that drugs were safe, kept thalidomide off the market in this country. Nevertheless, the episode led Congress to enact sweeping new legislation requiring drugs to be proven both safe and effective. This latter provision greatly extended the length of clinical trials and FDA review times, delaying the benefits patients receive from new therapies. In some cases, those delays prove fatal.
FDA often acts as though no cost is too great to bear in the search for absolute safety. And for nearly forty years, the single word, "thalidomide," has been enough to deflect just about any criticism of the agencys slow review process. Consequently, approval of the dreaded drug itself is an important symbolic act. But the new availability of thalidomide is cause for both celebration and despair. The news is certainly a medical breakthrough for victims of leprosy. It may also prove to be helpful for many patients suffering from dozens of other diseases. But a closer examination reveals the deeper flaw in FDAs methods.
Thalidomide was discovered to be effective in treating leprosy as early as 1965, and is now the standard medication around the world. It has been used successfully to treat lupus and some AIDS-related ulcers, and during the 1980s it became a common treatment in some countries for Kaposis sarcoma. Although its efficacy is not yet proven for other conditions, initial tests show great promise. Vicki Walton, who received the drug as part of a clinical trial to treat a rare immune disease, told FDA that it saved her life. "Without thalidomide, it would be back to where I waswheelchair-bound, bedridden, in the hospital."
Nevertheless, knowing that the approval process is long and expensive, no manufacturer or distributor was willing to request approval until 1996. FDA then spent nine months carefully examining medical research describing the drugs effects. It heard from both supporters and opponents of approval. Randolph Warren, a thalidomide victim who sits on FDAs thalidomide advisory panel, told the agency, "Ill never be happy until theres a world without thalidomide in it."
FDAs panel of medical and scientific advisors recommended approval in September 1997. Yet final consent was not granted until nearly one year lateran unusually long period of time even for FDA. Moreover, approval was conditioned upon very strong restrictions on distribution. Doctors and their patients must register with the drugs manufacturer and FDA before thalidomide can be prescribed. Women who take the drug must agree to use two forms of contraception and to submit to bi-weekly pregnancy tests.
Lost in the controversy surrounding thalidomides approval are the countless victims of FDAs one-size-fits-all regulatory scheme. How many patients suffered needlessly, waiting while FDAs chronic overcaution delayed the drugs US approval? Unfortunately, because Congress passed the FDA Modernization Act just last year, it is unlikely to revisit the issue any time soon. Worse still, a backlash against the recent reforms is now being orchestrated by such so-called consumer groups as Ralph Naders Public Citizen. Not only is further reform out of the question this year and next, the backlash threatens to reverse the small gains that were achieved.
The most important long-term goal is still to break FDAs approval monopoly. Let FDAs safety and efficacy standards remain as they are, but allow patients and their physicians to use unapproved drugs and devices with the clear knowledge that FDA has not certified them. Under such circumstances, FDAs fear of another thalidomide would no longer prevent sick patients from receiving potentially life-saving therapies. The agency might still be overcautious, but that overcaution would no longer be deadly.
Gregory Conko (firstname.lastname@example.org ) is a policy analyst with CEI.