Off-Label Prescribing of Drugs Calls
FDA Role into Question

Scott Esposito


The problem of FDA "drug lag" is widely recognized. New drugs are approved more quickly in Europe than the almost nine years it takes to get approval in the United States. Yet because drug recalls are just as rare in Europe as in the United States, Europeans seem none the worse for the shorter approval time; indeed, to the extent that Europeans can use new, health-enhancing drugs sooner, they are better off than their American cousins.

The European comparison shows that Americans would benefit from a streamlined FDA drug approval process. But, ironically, Americans can observe a safe, "unregulated" pharmaceutical market in their own backyard --- through the widespread practice of off-label drug prescriptions.

"Off-label" prescriptions are uses of FDA-approved drugs for purposes other than those approved by the agency. For example, aspirin, approved by the FDA as a pain killer, was used to reduce the mortality rate among heart attack victims for years before the FDA sanctioned that use of it. In his recent study of off-label prescriptions in The Independent Review (Summer 2000), economist Alex Tabarrok shows the rate of off-label prescribing is so high that most hospital patients receive at least one drug off-label.

Through the use of off-label prescriptions, doctors successfully determine the efficacy of drugs on their own and build up a community of knowledge. For example, when it was theorized that stomach ulcers were caused by bacteria, doctors tested this theory through the off-label prescription of antibiotics. As doctors discovered and shared information regarding the efficacy of antibiotics against stomach ulcers, amoxicillin and tetracycline were found useful in the treatment of ulcers without any FDA intervention.

The benefits of off-label prescribing are not limited to ulcers. Through off-label prescriptions the drug mitomycin, approved for the treatment of gastric and pancreatic cancers, has been found to be useful in the treatment of lung, bladder, breast, and cervical cancers as well as in certain forms of leukemia. Trimethoprim, a drug approved to treat pneumonia, has proven useful as an AIDS treatment.

In addition to discovering new treatments, off-label prescribing creates greater efficiency because it puts more choice in the hands of the public. Since off-label prescriptions are not tied down by FDA-sanctioned uses, doctors and patients are free to experiment with individualized treatment regimens.

Patients have clearly benefited from the freedom that doctors have to prescribe old drugs in new ways. Yet, the FDA does not allow doctors the same freedom to prescribe new but as-of-yet unapproved drugs.

The FDA claims that letting new drugs onto the market before they went through FDA-approved efficacy trials would be disastrous. The success of the unregulated off-label market shows that this claim is false. Drugs prescribed off-label have not been through FDA-approved efficacy trials but the process works so well that the Association of Community Cancer Centers, AIDS activists and several other organizations have lobbied health care providers to reimburse patients for off-label treatments. Many states now require insurance companies to do just that.

By circumventing the FDA, off-label prescribing allows for the more adept use of medication. The widespread use and success of off-label prescriptions shows that if the FDA loosened its regulatory grip and placed more trust in doctors, newer and more affordable drugs would reach patients faster and be better suited to their medical needs.

Scott Esposito is an economics and political science major at the University of California at Berkeley and a Public Affairs Intern at The Independent Institute, a public policy research and educational organization based in Oakland, California,

Originally published in the Medical Sentinel 2002;7(1):24. Copyright©2002Association of American Physicians and Surgeons (AAPS)