The FDA does not approve the uses of drugs

Many people believe that the Food and Drug Administration (FDA) of the United States approves uses of drugs. It does not.

This confusion comes from insufficient clarity of language.

The FDA does approve whether or not a prescription drug can be manufactured, marketed, and sold, the language on the label, and the claims of advertising.

Drug manufacturers must use an elaborate, detailed, lengthy, and very expensive process to bring a new drug to market. But, it is licensed physicians who determine, case by case, whether that drug will be prescribed, the dose, the dosage form, the route of administration, and the duration of use.

Drug manufacturers must pursue a similar process to achieve approval for any other uses for an already approved drug, for which they could market and advertise. They often do not, citing cost.

Many, even most, drugs are prescribed for uses for which approval for marketing has never been obtained.

The outward sign of approval, such as for what patient age or gender, is on the label. So the term “off-label” prescribing has evolved to refer to the practice of physicians prescribing for uses NOT on the label.

Premarket testing of many drugs has only been done on adults, sometimes only on white men. Use for women and children often is “off label.”

The same applies to uses of many cancer chemotherapeutic agents for different cancers.

This is a conundrum.

It behooves the drug companies to help physicians prescribe their drugs in the best ways possible. Physicians and patients as well as the company thus benefit.

But it is illegal for drug companies to promote any use that has not been approved by the FDA for marketing, and penalties for doing so are often steep.

Regarding “off-label” use, physicians can prescribe, patients can use, research can be done, journals can publish articles, books can include chapters, medical schools can teach, students and physicians can learn, CME credit can be granted, but pharmas are prohibited by law from supporting, advertising, or promoting such “off-label” use, directly or indirectly.

And, sometimes payers may refuse to cover therapies that are “off label,” even if it is the best treatment.