In the new Congressional $787 billion economic stimulus package, there is an earmark for $1.1 billion to compare different medical treatments for specific illnesses. This “comparative effectiveness research” will attempt to answer questions such as whether drugs or surgery work better in various medical conditions such as low back pain. According to the Washington Post, the bill would create a council of up to 15 experts to coordinate the research and advise the Government on how to apportion money.

The impetus for this program, endorsed by President Obama during his campaign, is a growing skepticism voiced by health economists and policy experts who feel much of what doctors currently do is expensive and doesn’t actually work. Some of the money will be used for clinical trials comparing different treatments and other funds will be used to analyze already published scientific studies. There is unquestionably much to be gained from such research. In many cases, surgical treatments haven’t been randomized against nonsurgical therapy and many drugs used in psychiatry haven’t been evaluated comparatively against non-pharmacologic treatments. In addition, there is insufficient follow-up on the long-term side effects of many approved drugs now on the market and many medical devices used today haven’t been sufficiently evaluated.

Comparative effectiveness research has generated great optimism in Washington. Hillary Clinton was one of the key advocates and Representative Pete Stark (D-Ca), chairman of the Health Ways and Means Subcommittee summarized the optimism thusly, “The new research will eventually save money and lives”. He explains the United States spends over $1trillion a year on health care and patients are put at risk with billions of dollars spent each year on ineffective or unnecessary treatments but “we have little information about which treatments work best for which patients”. In a report accompanying the economic recovery package, the House Appropriations Committee echoed Stark’s hopes by saying this research could “yield significant payoffs” because less effective, more expensive treatments “will no longer be prescribed.”

Unfortunately, from a medical standpoint, these expectations may prove overconfident. It is a fool’s errand to predict the outcome of medical research in advance - the results may not be what we expect. We want cheaper therapies to be better and, in fact, they often are. But in medicine, it simply doesn’t follow that cheaper is automatically better. What then? What does the Government do if research finds expensive back surgery turns out to be more effective than medication and physical therapy? What happens if long-term psychotherapy happens to be more effective at treating depression than short-term antidepressant therapy? What if cardiac surgery demonstrates it prolongs life for patients in their 80’s and 90’s? Will researchers feel subtle unstated pressure, or even overt pressure, to gear studies that will result in findings that allow the Government or insurers to limit coverage for expensive treatments?

This is a fundamental dilemma with large scale Government sponsored medical research. Quite often, the results depend on who does the studies. The make-up of the proposed council of 15 Government advisors will likely have a major influence not only on the type of studies but on the actual findings of those studies. This is not a theoretical concern. European countries have faced this exact problem translating Government sponsored comparative effectiveness research into public policy.

Completely disinterested researchers are not always those selected to perform studies. Some scientists may feel political pressure to turn out results sought by their patrons. Moreover, it’s the rare specialist or surgeon who performs a study and acknowledges his specialty’s approach is inferior to the alternative, especially if these findings have major financial implications for his specialty. Even then, professional specialty organizations are often loath to accept such findings. Anyone who doubts this, need only consult the nearest medical library.

All of this is likely to result in warfare between strange bedfellows. Consumer groups, unions, employers and insurers are likely to support the Government research efforts as a means of reducing waste. The for-profit health care industry will find itself on the other side of the trenches since programs to make health care more efficient may be viewed as a threat to their economic well-being. As health writer Maggie Mahar aptly puts it, “One man’s risky and over-priced treatment is another man’s income stream.”

Despite the best efforts of our best scientists who do comparative effectiveness research, there will always be uncertainty in medical treatment. Hopefully, the economists, policy makers and doctors remember that fact since billions of dollars and the nation’s health is at stake. They should heed Swedish physicians with extensive experience in comparative effectiveness research in Europe who caution, “A decision to prioritize a less therapeutically effective medicine because of cost-based considerations over an effective, but more expensive, medicine could lead to some serious political, social and moral dilemmas”.

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