Medicare Should Just Say No to Unproven Drugs

03/02/2009 05:12 am ET | Updated May 25, 2011

For a new administration committed to fixing America's health system by only paying for medicine we know works, Medicare's decision to pay for off-label chemotherapy drugs, ably described by the New York Times presents a challenging opportunity. Reversing this policy could send a powerful and positive message.

The new rule commits the government to paying for some anti-cancer drugs that the Food and Drug Administration hasn't found effective in dealing with the particular diagnosis involved. In other words, a drug the FDA has blessed to combat breast cancer would be used on someone with prostate cancer. All involved agree that these "off label" uses are experiments that often fail.

Despite the odds, they are popular. Patients like them because they provide a glimmer of hope in an otherwise bleak prognosis. Doctors want to expand their arsenal when they're exhausting their ammunition. And drug companies like them a lot because they modestly increase sales in the short run and provide the potential, if successful, of vastly increasing sales later.

But that's hardly a compelling argument for having Medicare -- or private insurers -- pay for them. From my perspective, it is an expensive investment in hope that's discordant in a system based on science. It raises some interesting and provocative questions about where to draw the reimbursement line and whether the goal of evidence-based medicine is achievable.

How far are we willing to stray from FDA's findings? If we're willing to use a drug for a different type of cancer, should we pay to use anti-cancer drugs on those who are not responding to conventional therapy after a stroke? And if that's okay, why rely on FDA approval at all? If someone wants to use a hyper-pure (and very expensive) peanut butter to help heal wounds, why discourage such experimentation?

It is all too easy to push this argument to absurd lengths. Given the fact that some patients are convinced they're alive today because of a visit to Lourdes when all other options had failed, do we have a responsibility to pay for visits to shrines when all relevant conventional medical remedies failed? Perhaps a society that was richer and more sensitive than contemporary America would do so, but we're not about to.

For my tax dollar -- and my health somewhat dependent on my Medicare coverage -- I'd go for a policy that had Medicare defer to the FDA and only pay for drugs used for purposes that agency had approved. That would limit experimentation, but would focus our medical investments on the areas where the probability of a positive payoff was greatest.

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