Prescribing for unlabeled conditions: patient benefit or therapeutic roulette?

There is ample evidence that prescribed medications are employed for uses far broader than the approved label indications in the U.S. An enormous research agenda thus exists that should be addressed in the not-too-distant future. In fact, it seems essential that operation of the Medicare Catastrophic Drug Benefit program be designed with the best available knowledge in this area. Perhaps it might be appropriate for several universities, the U.S. Pharmacopeial Convention, and/or the FDA to establish a center to study this question. This model has been applied with clinical/surgical registries, with adverse reaction reporting, and with device failures. We need a rational, science-compatible, and uniform policy free of political and emotional arguments to address the issue of handling, monitoring, and regulating the use of drugs for unlabeled conditions. Comprehensive data should be provided for policy makers, regulators, payers, and clinicians in their evaluating the use of different drug products. Even a brief glance at any page from the National Disease and Therapeutic Index shows intended use that would cause most experts to react in disbelief. Further, there seem to be relatively few instances in which the use of a given pharmaceutical for an unlabeled indication would qualify as a drug of choice in the first place. The therapeutic and economic consequences of the use of legend drugs for unlabeled indications are difficult to document. We do know that a significant proportion of hospital admissions and days can be traced to the inappropriate use of pharmaceutical products but the net impact of our subject on institution cost has not been established.(ABSTRACT TRUNCATED AT 250 WORDS)