April 21, 1999
Web posted at: 12:02 p.m. EDT (1602 GMT)

Much-awaited results were released last week from tests on a controversial but widely adopted treatment for breast cancer. "Standard" treatment calls for a course of low-dose chemotherapy, but researchers have been collecting data about whether high-dose chemotherapy and bone marrow transplant (BMT) work better. Some patients have successfully demanded that insurers pay for what has been an unproven therapy. But letting patients choose their therapy is controversial because BMT is substantially more expensive and creates much greater side effects than standard chemotherapy. The first results are not promising. Four of the five studies indicate no better survival rate for breast cancer patients who undergo BMT than for patients receiving standard care, and in the fifth study BMT patients had only marginally greater survival. The studies raise questions about how an unproven therapy became a common treatment, whether wide access to unproven therapies ought to be restricted, and how to decide who should receive them.

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Word of a radical new treatment for breast cancer created understandable interest among women whose poor prognoses gave them little hope. News that BMT might cure an often fatal disease spread through the networks of women belonging to breast cancer advocacy groups, facilitated by the Internet. The hope created by a treatment that might cure a fatal disease spread quickly, and women with breast cancer pursued BMT. The increased risks and sometimes severe side effects seem worth accepting when there are few good alternatives.

There are a few ways to get unproven therapies: research participation, insurance coverage, and paying out of pocket. Only a few patients can participate in research, and not many can afford the costs of treatment themselves. Insurers are reluctant to pay for treatments that are not standard and often will cover the costs of "experimental" therapy only in limited circumstances. So how did BMT for breast cancer become a treatment covered by some insurers even though it was unproven? High demand, too few slots for research participants, and the public perception that it could save lives all created a situation in which insurers responded to public demand without scientific support. But to justify using a new treatment, we must show that its benefits are at least as good as standard therapy, with no greater risks -- or we at least need to have a sense that this is so in an individual case, if not for all patients.

We need carefully controlled access to therapies like BMT for breast cancer until it becomes clear whether it is at least as good as standard treatments. If new treatments are no better than (but just as good as) standard approaches, should patients have access to them?

New treatments first must be shown to serve the best interests of current and future patients before they can even be considered for wide adoption. This assessment includes not only how well they work, but what risks and side effects they bring. Only after fully weighing and balancing risks and benefits can we discuss whether new treatments are worth paying for, and by whom. Health care and biomedical research dollars must always be spent responsibly and wisely -- especially as managed care tightens access to health care resources, and demands on research funding increase. Adopting expensive but questionable and unproven treatments not only flies in the face of these responsibilities but sidetracks and undermines our real motivation: quality medical care that will save lives and improve health.