What the FDA s Decision on Thalidomide Really Means

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LOST IN ALL OF THE HULLABALOO surrounding the FDA s reversal last week of its 1962 ban on Thalidomide, the drug that was linked to horrific birth defects, was perhaps an even greater story: what essentially amounts to the very refocussing of the FDA s raison d etre.

When the FDA reversed itself and approved Thalidomide, there was no suggestion that the teratogenic results of Thalidomide would no longer occur. Rather, the move signals a changing of the paradigm that guides the agency.

Once burdened with the responsibility of protecting every American from potentially dangerous products, now the public and their physicians are being given a greater role in the decision making process. Under this arrangement, citizens are not only going to benefit from technology, they will also be well protected by responsible and limited safeguards.

The FDA s approval of Thalidomide was, at least officially, meant as a therapy for leprosy, an almost non-existent disease in this country. But with a wink and a nod, the agency s move gives doctors the green light to prescribe the drug to patients with serious diseases such as AIDS and cancer, implicitly permitting, even encouraging, such off label use. (The FDA could not directly approve Thalidomide for AIDS patients because there is no controlled data on its ability to help them.)

Doctors already have many potentially teratogenic therapies at their disposal (i.e. accutane, cancer chemotherapies), and as a society, we place our trust in their ability to prescribe them appropriately. These implicitly dangerous products, when not abused, have significant medical benefits. Why, then, should Thalidomide be judged differently?

Americans should welcome rather than criticize the fact that the FDA will no longer seek to remove choices that when abused cause harm, yet when used properly, can yield life saving benefits.

A recent study published by the Journal of the American Medical Association cites adverse drug reactions (ADR s) as a leading cause of premature death in this country. That study was flawed, however, because while considering ADR s, it did not take into account the net positive effect of our pharmacopoeia.

Every therapeutic intervention between a physician and a patient requires a balancing of risks and benefits. There is no such thing as an entirely risk free drug. Unfortunately, television coverage of Thalidomide s approval played to the bias against drugs, as illustrated in JAMA. While viewers saw graphic footage of Thalidomide s deformed victims, there was no video of the deathly ill patients for whom Thalidomide just may be a godsend.

Since the rigorous controls established by the FDA and the manufacturer are so strict, and the potential harm so well known, the horrific effects for which Thalidomide became notorious can only befall a pregnant woman under the most reckless misuse. Talking heads are suggesting that if there is just one adverse reaction, the FDA should reconsider the approval. This zero risk approach is senseless. The FDA, aware of Thalidomide s risks, made the right call. If there is an adverse effect, the blame should not be placed on the FDA, nor the drug, nor the manufacturer but on the failure of the prescribing physician and his or her patient to adequately appreciate the risks.

Why, indeed, should the wanton behavior of the few harm the many potential beneficiaries?

By approving Thalidomide, the FDA showed compassion for seriously ill patients who will benefit from its off label use. We optimistically see the FDA implementation of unprecedented safeguards for the use of Thalidomide as a sign that the agency accepts the risks involved with drugs and their off label uses.

The Food and Drug Administration is to be applauded for having the courage to reverse it s long standing policy against Thalidomide, thus marking a change towards a less intrusive, pro technology FDA.