In order to protect the nation from harmful or worthless drugs and devices, the Food and Drug Administration (FDA or the Agency) is legislatively authorized to restrict the sale of prescription drugs or medical devices to those whose efficacy and safety have been reviewed and approved by the Agency.1 Drugs and devices are approved for a specific medical purpose. In numerous instances, however, the medical profession has discovered that treatments approved for one purpose may also serve other valuable medical purposes.2 Indeed, on a number of occasions such "off -label" treatments have proven to be essential to the successful treatment of some very serious illnesses.3
In these off-label situations, the FDA is faced with a dilemma. On the one hand, off-label use of prescription drug and devices gives rise to a series of major problems for the FDA. While the drug and devices in question have been vetted and approved by the FDA for their designated purpose, at no point has the FDA reviewed the supporting scientific data to determine efficacy for the off-label purpose. It is therefore at least conceivable that if such off-label uses are permitted, the drug or device may in reality be worthless or even dangerous for its alternative use, yet doctors may be freely employing it for that purpose. Moreover, widespread off-label use of prescription drugs and devices conceivably undermines the FDA's authority and deters manufacturers from seeking on -label FDA approval for even widespread alternative uses. The fear, then, would be that the FDA's initial approval could serve as a wedge to permit the industry's equivalent of the Wild West, where the rule of law was seen only rarely. It is true that the Federal Government could avoid this danger by categorically prohibiting all off-label uses, instead requiring drugs and devices to gain administrative approval for each use. But this alternative is also not free from problems. As a result of such a ban, potentially valuable treatments- often for the most serious of diseases- could be tied up in years of administrative red tape while their manufacturers seek FDA approval. Even worse is the possibility that manufacturers might decide that seeking FDA approval for the off-label use is simply not worth its costs. In any event, under the current regulatory framework, the FDA asserts that it lacks legal authority to restrict the ability of doctors to prescribe drugs or devices for off -label uses.
The FDA has sought to resolve this dilemma by effectively swimming half way across a river- thereby pleasing no one and avoiding none of its quite legitimate concerns. It has resolved its dilemma by leaving off-label uses essentially unregulated, but to make a manufacturer's promotion of an offlabel use categorically illegal. As a result, while the benefits of off -label use are roughly preserved, whatever dangers might accompany such uses are in no way avoided since off-label uses are still generally allowed. At the same time, while it is true that the ban on off-label promotion by manufacturers prevents false or misleading advertising, because the ban is all-inclusive it simultaneously prevents promotion of valuable off-label uses of which doctors otherwise may well be unaware. To be sure, those other than the manufacturer are still permitted to discuss the relative merits of off -label uses, and even manufacturers are authorized to speak about certain circumstances surrounding off-label use, such as accompanying dangers. But the FDA could not have stated more clearly that manufacturers are prohibited from advocating off-label uses in any way. This is so, even where the FDA in no way challenges the accuracy, truthfulness or completeness ofthat promotion.4 As a result, in many instances doctors are likely to be deprived of valuable information about important off-label uses that are totally lawful and extremely beneficial to some very sick people. In short, the FDA's policy, as well intentioned as it no doubt is, has made a shambles of off-label use, by simultaneously over-regulating and under-regulating the practice. …