Is Medicare backpedalling on evidenced-based medicine?

Note: This post first appeared at

Both the New York Times and the Wall Street Journal
carried stories today on Medicare's expansion of the number of
drug-listing compendia that can now be used to justify reimbursement
for the off-label use of anti-cancer drugs. This expansion, which
GoozNews covered last summer (see posts here, here, and here), will sharply increase Medicare spending on anti-cancer drugs of questionable medical value.

It has also provided drug companies with an alternative system for
getting reimbursed that won't require their going to the Food and Drug
Administration to prove that the regimens listed in the compendia
actually benefit patients.

The effects of this new system were understated in the articles. The Times
estimated that the higher spending by Medicare will come on top of the
$2.4 billion the senior citizen health care program spent on cancer
drugs in 2007. But according to this 2006 testimony
by Center for Medicare and Medicaid Services official Herb Kuhn,
Medicare spent about $10 billion on "Part B" drugs in 2005 (these are
drugs administered in physicians' offices, which includes most cancer
drugs), and about half of that went to oncologists.

The Times also buried near the end of its story (and the Journal
ignored) revelations about the most significant conflict-of-interest
affecting how drugs get off-label indications listed in the compendia.
It involves the oldest and traditionally most trusted of the compendia
— the American Society of Health-System Pharmacists' drug information
formulary or AHFS-DI.

In late 2007, the cash-strapped trade group for in-hospital
pharmacists hired a misnamed non-profit called the Foundation for
Evidence-Based Medicine to serve as a go-between for drug companies
that wanted to get off-label uses of their drugs into the compendium.
If companies gave the Foundation $50,000, it in turn would provide
AHFS-DI with the resources to conduct a review.

AHFS-DI had come under fire from oncologists and drug companies for
being slow to approve off-label uses. Indeed, that was the reason
offered by CMS for wanting to expand the number of allowable compendia
in the first place.

Gerald K. McEvoy, who runs AHFS-DI, told the Times that the
foundation "insulates the guide’s staff from industry pressure" and
that "fewer than one-third of the reviews under the new arrangement
have resulted in a positive recommendation in the compendium."

That statement is disingenuous. You don't need to be "recommended"
to get reimbursement for an off-label use. Medicare's regional
contractors, who make payment decisions in the absence of national
coverage decisions, have discretion to pay for drugs even when the
evidence is equivocal. When presented with payment requests from
community oncologists, most do. And virtually all the most recent
listings in AHFS-DI under the new system (see the most recent listings here) received at least an equivocal rating.

Moreover, McEvoy's statement ignores the most pernicious aspect of
the system. A pay-to-play listing system — just like the Prescription
Drug User Fee Act at the FDA — allows the drug industry to determine
what off-label uses will get evaluated. Think about the poor academic
researcher who conducts and publishes a clinical trial that shows an
innovative use for a generic chemotherapy drug that has been around
since the 1950s. Who will pay the $50,000 to AHFS-DI so that use gets
listed in the compendium?

Beyond the conflicts of interest now driving both oncologists
decisions about and Medicare's payment for anti-cancer drugs, the more
serious long-term issue in this government-encouraged expansion of
off-label use revolves around Medicare's refusal to collect outcomes
data. Again, the issue was addressed in a single easy-to-miss paragraph
in the Times story that is worth highlighting:

“We have very little faith that those indications that make
it into the compendia are safe, let alone effective,” said Dr. Allan M.
Korn, the chief medical officer for the Blue Cross and Blue Shield
Association, who added that Medicare should cover off-label drugs only
if the results of their use are carefully tracked afterward. There is
no such requirement in the new Medicare guidelines.

In the stimulus bill now moving through Congress, both houses have
set aside more than $20 billion to encourage physicians to adopt
electronic medical record keeping. It includes hefty tax incentives for
physician offices and hospitals to adopt the new systems. Doesn't it
make sense to also require that physicians who get those incentives,
including oncologists, collect outcomes data from the off-label use of
drugs and report that information, properly protected for patient
privacy, to paying agencies? How else are we going to learn if all the
"hope" that taxpayers financed for dying cancer patients actually
turned out to have helped?

The one thing we can be sure of is that the less-than-rigorous
clinical trials that lead to compendia listings, often evaluated by
compendia committees that have close ties to the very companies that
make and sell the drugs, do not amount to evidence-based medicine, no
matter how many foundations with that name the compendia-writing
organizations set up.

Merrill Goozner has been writing about economics and health care for many years. The former
chief economics correspondent for the Chicago Tribune, Merrill has
written for a long list of publications including the New York Times,
The American Prospect and The Washington Post. His most recent book,
"The $800 Million Dollar Pill – The Truth Behind the Cost of New Drugs
" (University of California Press, 2004) has won acclaim from critics
for its treatment of the issues facing the health care system and the
pharmaceutical industry in particular. You can read more pieces by Merrill at,where this post first appeared.

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2 replies »

  1. In the setting of metastatic cancer, if you look at the NCI-recommended treatments, you’ll find that all patients in this setting are candidates for clinical trials of some sort. This is just another way for the NCI to say that there is no single “best regimen” to give to the “average” cancer patient and it is therefore entirely appropriate to consider novel drugs or drug combinations for different types of cancers.
    It has been very routine and well-accepted practice to prescribe drugs in cancer types and disease stages outside of those in which the drugs originally received FDA approval. Generally, however, insurance companies have paid for drugs used outside of FDA-approved settings because the treating physician finds their use in those instances to be “medically necessary.”
    Medicare has radically expanded its authorization for use of cancer drugs by putting off-label decision making in the hands of compendia writers in the private sector, many of whom are on the payrolls of the companies that make the drugs. An estimated 60 percent of anti-cancer drugs are used off-label.
    I’m all for cancer patients, particularly at end-stages, receiving these needed drugs. My personal belief in having additional support of drug patient-specific activity, as determined by extensive laboratory pre-tests to improve patient outcomes, could very well bolster an argument for off-label use of specific cancer drugs, with no economic ties to outside healthcare organizations; recommendations made without financial or scientific prejudice.

  2. this has already trickled down on private insurers, we use to be able to deny coverage with the excuse it wasn’t medicare approved, now that they cover every hunch and long shot it’s harder for us to deny questionable care. How do we go to court and say we don’t think this off label treatment is justified when they hold up Medicare reimbursement as proof it is. Not only have they greatly increased the expenditures of Medicare but also private insurance.