Comparative Effectiveness Research Cornered by Foes

Can $1.1 billion make comparative effectiveness research a regular part of medical care and health insurance in the United States?
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That's how much the 2009 stimulus bill — the American Recovery and Reinvestment Act — devoted to this type of research, which aims to produce better information about the costs and benefits of alternative treatment options. It differs from clinical trials that compare new drugs to placebos and treatments to control groups. It instead compares multiple treatments, evaluating both effectiveness and cost. When done well, it can both improve treatment and save money.

But that may not be enough. Although reliance on comparative effectiveness research seems eminently reasonable, opposition comes from diverse sources. Advocates in Congress managed to overcome resistance to including funds for it in the stimulus bill, but making use of the research will be harder.

Physicians often have trouble using research statistics to explain the value of new treatments to patients. (See "Convincing the Public to Accept New Medical Guidelines.") In addition, however, opposition organizes around views on health policy.

Conservatives opposed to national health insurance view comparative effectiveness research as a step toward rationing. They worry that the government will draw on it to decide what treatments a patient can or cannot get. As Newt Gingrich says in a Wall Street Journal commentary, a panel requiring research evidence would restrict choices: "We do need basic health reforms. But their focus should be on maximizing patient choice and freeing health care providers."

Conservative political commentator and columnist Michael Barone states his opposition more strongly: "But comparative effectiveness research is, if not junk science, not a fully developed intellectual enterprise. Medicine is an art as well as a science, and comparative effectiveness research may too often compare apples and oranges."

Liberals have their own concerns. Some believe that health insurance companies can use comparative effectiveness research to deny expensive but needed treatment to patients. Private insurance companies have stronger incentives to use (or perhaps misuse) it in ways that increase profits than to improve treatment. In principle, it could increase costs when expensive treatments prove superior. With health care based on profit rather than a government single-payer system, however, private insurers may favor the inexpensive options.

Physicians rely extensively on medical research, and the American Medical Association supported comparative effectiveness research funding in the stimulus bill. However, some physicians worry that research findings may overrule their clinical judgments. Statistical results that generalize across groups may not fit the individual problems and circumstances of patients. Critics say that it could produce cookbook approaches to treatment and threaten personalized medicine.

Elderly Medicare recipients express some of the same worries. Larger Medicare reimbursements for the most effective and cost-efficient treatments would provide incentives to save the program billions. Despite assurances to the contrary, however, some elderly participants believe that using this research to cut costs will further restrict their choices. Legislators insisted on including wording in the stimulus bill that prevents use of comparative effectiveness in determining coverage and payments.

Pharmaceutical companies see other reasons for concern. They support comparative effectiveness research in principle, but the specifics could bring challenges. Their prescription drug products currently go through scientific analysis for safety and effectiveness before the FDA gives approval. These studies must pass a traditional standard for approval: The drug must work better than a placebo. Yet, advocates of this research want something more. New drugs should work demonstrably better than existing — often older and much cheaper — drugs to treat the same condition.

Blockbuster drugs now rely on huge advertising budgets to spur sales. Yet, findings that an expensive new drug works no better than a cheaper alternative sold in generic form won't spark an exciting advertising campaign.

Who's left to push hard for the goals of comparative effectiveness research?

Mostly, it's academic researchers and those in Congress, the government and insurance companies wanting to save money. But appeals to saving money lack the power to counter what Dr. Jerry Avorn at Harvard University calls an avalanche of non-facts used in opposition to this type of research. The public may favor lower health costs in general but not when it leads to what they view as limits on their treatment options.

Defense of comparative effectiveness research needs something more than concern with the bottom line. Writing in the New England Journal of Medicine, Avorn suggests another strategy: Make a moral case for the value of knowledge and the leadership of America in generating that knowledge. Knowledge is a public good like clean air and water. Practitioners, patients and policymakers can benefit from it.

For example, a famous 2002 study of 30,000-plus patients with high blood pressure found that inexpensive diuretics worked as well or better than much more costly drugs. Rather than restrict choices, the knowledge gained from the study gives physicians and patients help in selecting wisely from the many choices available.

Indeed, knowledge can improve personalized medicine. Drs. Alan Garber and Sean Tunis argue that this research often produces findings on how treatments benefit some patients more than others. The knowledge can help match treatment to the needs of individual patients. It can make the American health care system better.

Problems exist in comparative effectiveness research. For one, experts need to translate research findings into clear and usable clinical recommendations that physicians and patients can use. For another, they need to package the recommendations in ways that complement rather than replace clinical judgments.

Still, the guiding principle that knowledge is better than ignorance remains powerful. Despite their varied concerns, conservatives, liberals, medical practitioners, insurance providers, pharmaceutical companies and patients can agree on that.

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