The new health care bill passed by Congress in March calls for increased funding for so-called comparative effectiveness research. These studies, which directly compare different treatments and strategies against each other, address fundamental questions such as: Which of the more than 30 available blood pressure medications is best? Is counseling as effective as medications for the treatment of depression? Do patients treated in large hospital-based practices fare better than those in small practice settings?
With medical decision making becoming increasingly complex, we believe the need for comparative effectiveness research is self-evident.
But not everyone agrees. Following the passage of the new bill, there has been an outpouring of criticism of comparative effectiveness research. One recent piece in the Wall Street Journal asserts that comparative effectiveness studies focus on "analyses of older, previously completed studies" and therefore "is unlikely to help [patients] in 2010." Another in the LA Times criticizes efforts to require pharmaceutical companies to compare new therapies with alternative treatments rather than placebos, arguing that such requirements might make drug research too expensive or risky for pharmaceutical companies. Others warn that comparative effectiveness studies threaten "personalized medicine" -- in which doctors use clinical judgment to tailor treatments to individual patients -- by denigrating therapies that prove inferior in large studies but might be effective among certain patients. Some also fear these studies could be used by insurers to deny coverage for expensive therapies that prove no better than cheaper alternatives.
While these concerns must be taken seriously, we believe they are based on fundamental misunderstandings about comparative effectiveness research.
First, the claim that comparative effectiveness research involves outdated information is simply not accurate. Comparative effectiveness research differs from non-comparative studies only with respect to the types of questions they address. There is no reason to suspect these studies will yield older data. We can attest that the vast majority of comparative effectiveness studies in a recent analysis of comparative effectiveness research that we co-authored in the Journal of the American Medical Association involved highly relevant therapies that are used every day in clinical practice.
Second, while we acknowledge that requiring drug companies to compare new medications with existing therapies will require a shift in how companies study their drugs (our recent analysis showed that two-thirds of commercially funded drug studies use a placebo comparator), such a requirement should actually encourage innovation, not stifle it. According to the Food and Drug Administration, approximately 80% of newly approved medications are "me-too" drugs that offer no clear benefits over existing ones. If companies were required to compare new therapies with existing ones, it might promote the development of novel classes of medications that offer real advantages.
Third, concerns that comparative effectiveness research could threaten personalized medicine are off-base. In fact, the opposite is true: the Federal Coordinating Council on Comparative Effectiveness Research -- which will oversee the new federal comparative effectiveness funding -- has repeatedly highlighted the importance of research that examines therapies in different populations such as the elderly, children, and under-represented minorities, as well as for research that will help doctors tailor therapies using genetic information. Ironically, it is comparative effectiveness research, not placebo-controlled trials, that will help doctors personalize therapies.
Fourth, although insurance companies may use the results of comparative effectiveness studies to deny coverage for new therapies that prove inferior to existing ones, in most instances these denials will be in the patient's best interest. It is likely that many patients currently receive treatments that are less effective than alternatives, but without comparative effectiveness studies, no one knows which they are. Furthermore, comparative effectiveness research may actually be used to justify therapies that are not effective in the population as a whole but work among a subset of patients.
While we acknowledge the important concerns about comparative effectiveness research, we are confident that the benefits of these studies will far outweigh any downsides. In order to ensure that the U.S. remains the world leader in medical innovation, we cannot shy away from comparing the best existing therapies and strategies head-on.
Dr. Michael Hochman, MD, is an Assistant Clinical Professor of Medicine at the Keck School of Medicine, University of Southern California. Dr. Danny McCormick, MD, MPH, is an Assistant Professor of Medicine at Harvard Medical School. They coauthored an analysis of comparative effectiveness research, "Characteristics of Published Comparative Effectiveness Studies of Medications", published in the March 10 edition of the Journal of the American Medical Association.