Re: Oncologists Judge Themselves the Best Judges of Cancer Treatments

Extract

Ms. Napoli makes a serious error when she refers to “off-label” drug prescription as the use of drugs “in instances when efficacy is unproved” (1). The U.S. Food and Drug Administration (FDA) is not in the business of cataloguing all of the “proven” uses of drugs. Its responsibility is to determine that a drug is safe and effective for at least one indication before it can be marketed by a pharmaceutical company. Labeled uses are merely those that the FDA allows the manufacturer to state as indications based on the information provided by the manufacturer. The FDA's authority is limited to monitoring the marketing practices of pharmaceutical companies. It has always, appropriately, left decisions about the proper uses of approved drugs to clinicians.

Unfortunately, many third-party payers share Napoli's view. They do not understand that, if a drug is proven to be useful for a new indication, its labeling will not change to reflect that, unless the manufacturer decides to go to the substantial expense of presenting evidence to the FDA to request it. Furthermore, there are many instances in which a drug can be reasonably expected to be useful even in the absence of “proof.” An example of this situation involves the treatment of rare cancers. Extrapulmonary small-cell carcinomas share biologic and clinical features with their pulmonary counterparts and are therefore widely recognized to be appropriately treated with similar drug regimens (2). Likewise, bile duct carcinomas are histogenetically similar to pancreatic cancer and might be expected to respond to treatments that are effective for the latter tumor. Yet both of these scenarios are so rare that they will never be subjected to evaluation by randomized, controlled trials. Furthermore, they would be unlikely to head any pharmaceutical company's list of priorities for obtaining approval for FDA labeling.