Thalidomide Without Tragedy

Few drugs have caused more wrenching birth defects than thalidomide, which is why the Food and Drug Administration banned it in the 1960s after it was used as a sedative. A number of children born to women who took thalidomide during pregnancy had short “flippers” or no limbs at all; many survive today, testaments to the substance’s awful power.

This week, the FDA announced its intention to legalize thalidomide again, this time for use on leprosy patients, for whom the drug has proven highly effective.

Heated debate is expected in Congress, where some legislators are already questioning the FDA’s decision. But in the end, legalization should proceed despite the likelihood that use will spread beyond leprosy, a relatively rare condition in the United States. According to U.S. law, once a drug is approved, physicians can prescribe it legally for “off-label,” or officially untested, uses. Studies have suggested that the drug could be highly effective for certain cancers and advanced AIDS.

To ensure that thalidomide is used sensibly, the FDA has proposed registering recipients in a national database. It would also require women of childbearing age to show proof that they were using contraceptives before they could be considered for treatment.

The national database would allow the FDA to track the type of illnesses for which thalidomide was being prescribed. However, if the agency identified uses it suspected were dangerous, it would be unable to prohibit them. Congress should give the FDA special authority to halt certain off-label uses until the manufacturer completes safety tests. Moreover, Congress should delete provisions in two pending FDA reform bills that allow Celgene, which is the marketer of thalidomide, and other drug makers to promote off-label uses of their drugs.

If legalization, as expected, is approved, an official at Celgene says the company is “ready to roll” to meet expected demand. Congress, however, must give the FDA the authority to roll just as swiftly to ensure that this most promising and dangerous of drugs is used with extreme caution.