Prescribing Off-Label:
Cutting-Edge Awareness

By Frances Maguire Paist, Staff Writer

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.

  •  The use of off-label anti-cancer therapies has increased since 2000.

  • The oncologists interviewed confirm that while they do look to drug summaries for reimbursement guidance, they rely heavily on peer-reviewed literature and other broader reference sources for clinical decision making.

The Covance study emphasizes that cancer treatment is in an exciting era, one in which rapidly-advancing technology can often improve the length and quality of life for patients. Sometimes the results come much too quickly for the drug summaries and other approved communication vehicles on which the insurance industry has based its coverage decisions. And while most oncologists are aware of cutting edge treatments almost immediately, some are hesitant to offer them because of the uncertainty of reimbursement. Says Dr. John Feldman of the Regional Cancer Center in Greensboro, North Carolina, “The question is not evidence-based medicine but what is acceptable evidence and who should be the judge of that information.” Feldman continues, “Reimbursement rules are unclear at best, and there are extremely long delays in payment even when claims are successful. Unfortunately, many oncologists, in the end, feel it is easier and less risky to avoid offering these new therapies than to jump through the many hoops required to recover money already spent on the drug in question.” Indeed, cites the study, there is a profound effect of the red tape hassle on the practice of oncology and on the newest treatments available to patients.

One of the goals of the study was to determine if Medicare or private insurers were creating barriers to the use of off-label anti-cancer therapies. Forty-three percent of oncologists interviewed said their use of off-label anti-cancer therapies is increasing for reasons like more aggressive cancer treatment and narrower FDA labeling on approved drugs. However, 30 percent said their off-label use is decreasing. Of that audience, 33 percent cited reimbursement as an issue. Fifty-four percent of the oncologists interviewed said that Medicare coverage issues frequently or very frequently cause them to alter treatment decisions. Twenty-nine percent indicated that private policies have had this effect on them. Since 1991, a law has been in place requiring Medicare to cover off-label use of anti-cancer therapies if it is supported by drug compendia or peer-reviewed literature. So while the law is in place to help doctors and patients alike, more progress needs to be made on what is considered proper support documentation.

The Covance study concludes by saying that peer-reviewed literature (on which oncologists rely heavily) should be accepted as a basis of coverage for off-label use in addition to drug compendia. The study states that listings in drug compendia are often outdated or incomplete and may not include off-label uses for potentially new drugs that might be supported by other published clinical evidence. Future policies, they say, should be written to encourage off-label use of anti-cancer therapies and to reduce the administrative burden on oncologists seeking to find out if patients are covered for a certain drug. Medicare carriers should make clear what is required in order to qualify for coverage of off-label use of anti-cancer therapies, and documentation requirements should be minimized for anti-cancer therapies used regularly for rare cancer types.

But wow. In a world of hurt, the last thing a cancer patient wants to know is that a decision about whether or not a treatment will be covered by insurance might take precedence over its efficacy. The Declaration of Geneva, which has for some medical schools replaced the Hippocratic Oath, states, “The health of my patient will be my first consideration.” First. Not last. Not sometimes. Patients invest a great deal of faith and trust in their oncologists and depend on them to make healing decisions that should have nothing to do with money. That oncologists are people too with businesses to run is a fact of life. That we patients humbly ask them to be more is part and parcel of their having chosen to be a healer. 


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