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Prescribing Off-Label:
Cutting-Edge Awareness

It has all the elements of a riveting read: politics, money, illness and conscience. Questions abound that encourage the reader to proceed with caution. At the end of the day, though, perhaps because some physicians as well as insurers are key players, readers who have ever been treated for cancer will simply be stunned.
Using drugs outside of their approved regimen and prescribed course of treatment is called off-label use. Since the early 1960s, the Food & Drug Administration (FDA) has been given wide authority by Congress to assess the safety and effectiveness of all drugs before they can be marketed in the United States. The European thalidomide tragedy in which approximately 12,000 severely deformed babies were born to mothers who had taken the approved tranquilizer as a sleeping aid or for nausea control during pregnancy precipitated this.

Here’s the drill – once a drug has been approved by the Food & Drug Administration and language regarding its dosage recommendations finalized, the FDA does not attempt to regulate the practice of medicine. Indeed, it is the physician who makes decisions about a medication’s use, and choosing to use one on an off-label basis is quite legal. The world where off-label usage of drugs is most commonly seen is in cancer care. Oncologists, physicians specializing in cancer treatment, are often charged with deciding how and when to use a drug in a specified manner.

Even though pharmaceutical companies who learn their drug is being used in an off-label manner are supposed to report it, this rule is not enforced by the FDA. Certainly it takes time and money to have drugs approved for specified purposes, and in our rapidly-changing world replete with cutting edge cures presenting in rapid-fire fashion, it’s a win-win proposition for all when remedy can take precedence over red-tape paper shuffling. In this way, pharmaceutical companies get much-needed drugs to market that provide cutting edge cures, physicians heal, and patients get well.

But enter insurance. It is here where the story becomes complicated, here where the reader will be stunned, here where knowing the numbers will add necessary caution to the results. When it became known that there were certain instances in which the off-label use of drugs was not approved for coverage by certain insurers (most notably Medicare), and because it was suspected that certain physicians allowed treatment protocols to be influenced by whether or not they would be covered by insurance, three groups decided to fund a study called “The Impact of Payer Coverage and Reimbursement Policies on Physician Off-Label Use of Anticancer Therapies.” The recently released results (September 2005) have revealed interesting information that promises to be studied further; however, it should be noted that the study was quite small, including only 28 oncologists and 12 oncology practice managers. While results obtained from such a small study, even though geographically diverse, are interesting, it is unlikely that they can be called statistically significant. After all, there are thousands of oncologists and oncology practice managers nationwide, and so this small sampling is just that – a sampling.

The results of the study conducted by Covance show:

Sixty-eight percent of interviewed oncologists state that off-label use of anti-cancer therapies plays an important role in cancer treatment. Twenty-one percent cite medium importance. Ninety-three percent of oncologists interviewed reserve off-label use of anti-cancer therapies for those suffering with advanced stages of cancer.

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