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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