An analysis by researchers at the Keck School of Medicine of the University of Southern California (USC) has found that only 32 percent of medication studies published in top medical journals compare the effectiveness of existing treatments. These studies, known as comparative effectiveness studies, help doctors know which therapies work best and under what circumstances they are most effective.
More government funding, and other changes, are needed to promote comparative effectiveness studies and improve their quality, according to the study's co-authors, Michael Hochman, M.D., assistant professor of clinical medicine at the Keck School, and Danny McCormick, M.D., M.P.H, assistant professor of medicine at Harvard Medical School.
The study, "Characteristics of Published Comparative Effectiveness Studies of Medications," published in the March 10 edition of the Journal of the American Medical Association (JAMA), provides the first formal analysis of comparative effectiveness research, which is a priority issue for the Obama Administration. Last year, Congress appropriated $1.1 billion in funding for comparative effectiveness studies as part of the American Recovery and Reinvestment Act.
"Research on new therapies is, of course, critical for scientific advancement, but we also need research that examines how to use existing therapies appropriately," said Hochman, lead author of the study. "For example, which of the more than 30 blood pressure medications on the market works best and in whom? Are certain diets and exercise regimens as good as medications for controlling cholesterol? Is it safe to aim for normal blood sugar levels when treating patients with diabetes? These are questions that comparative effectiveness studies should address."
Hochman and McCormick analyzed 328 medication studies published between June 1, 2008 and Sept. 30, 2009 in six leading medical journals, of which 104 (32 percent) were comparative effectiveness studies, as defined by Hochman and McCormick's analysis. The rest either compared medications against an inactive control group (such as a placebo) or involved unapproved therapies not currently available to doctors.
The study also showed that just 11 percent of the comparative effectiveness studies compared medications with non-pharmacologic therapies (such as lifestyle changes or surgery), and fewer than a third compared different medication strategies (such as the optimal blood sugar target in patients with diabetes). The rest compared medications with each other. In addition, just 19 percent of the comparative effectiveness studies focused on safety and only 2 percent included cost-effectiveness analyses.
"Most of the comparative effectiveness studies we reviewed simply tested whether medication 'x' is better than medication 'y,' rather than addressing fundamental questions such as 'How can we use this medication more effectively? When is this medication better than surgery? Which among two effective approaches is the safest?'" said McCormick, the study's senior author.
The research also showed that 87 percent of the comparative effectiveness studies received at least some funding from non-commercial organizations, such as non-profit foundations or government institutions.
"Despite the widespread belief that the pharmaceutical industry conducts most clinical research involving medications, our study shows a critical need for non-commercial funding for comparative effectiveness research," Hochman said.
Hochman and McCormick believe their results highlight the need for more government funding for comparative effectiveness research. In addition, Hochman and McCormick recommend that the U.S. Food and Drug Administration require pharmaceutical companies to compare medications with other existing treatments whenever alternative therapies for a particular condition exist. Currently, companies often win approval for their products simply by showing that they are better than a placebo.
"Many of our nation's research priorities are driven by the pharmaceutical industry," Hochman said. "The pharmaceutical industry, not surprisingly, focuses on the development of new and marketable products. But once these products win approval, we need funding for research that examines how to use these therapies in an effective, safe, and rational manner."
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