The forward momentum of medical progress is manifest, it could be argued, in the $50 billion spent in 2008 on pharmaceutical research and development in the quest to bring new drugs to market. But little scientific or governmental infrastructure exists to ensure that each new treatment is actually an improvement over existing therapies—and to tease out what therapies are best for which patients.
People facing tough medical decisions, such as cancer patients, often have to work with their doctors to decide what combination of surgery, chemotherapies, radiation, lifestyle changes or other treatments will likely be the most effective choice. The array of options can be dizzying and most patients, and even some doctors, are ill-prepared to do the background research to glean an answer. And in many cases there is often no data available that compares the effectiveness of various drugs and other treatment options.
Support for this sort of work, known as comparative effectiveness research (or CER, which is defined by the federal government as comparison of "the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions"), has begun to spread from sectors of the scientific world into the public and political realms—thanks in part to health care reform debates and $1.1 billion in funds from the 2009 American Recovery and Reinvestment Act (ARRA).
But a new review of recent literature reveals that medical research has shied away from direct comparisons of therapies. In fact, less than a third of recent studies published in the top six medical journals could be classified as CER, according to the new report, published in the March 10 edition of JAMA The Journal of the American Medical Association.
Comparative assessment might not be as sexy as discovering the next blockbuster drug—and is not likely to ever supplant development—but it is a crucial aspect of patient care. "As we develop more and more treatments, it's important that we know we can use them in an effective, rational way," says Michael Hochman of the Keck School of Medicine of the University of Southern California and lead author of the report. He found that many of the published studies failed to assess "what we considered to be fundamental therapeutic decisions."
The knowledge gap between new discoveries and best treatments "is frustrating for patients, who too often undergo trial and error medicine," Patrick Conway and Carolyn Clancy, both of the U.S. Department of Health and Human Services (HHS), wrote in an editorial accompanying the new study. "The United States leads the world in biomedical science," they noted, but it "now must implement a framework for research that advances patient-centered care"—care that is based not just on more options but a better understanding of these options, they asserted.
Comparing the comparative
When a new treatment hits the market, it is often accompanied by a wave of promotion (aimed toward doctors—and sometimes patients themselves) in addition to the trial results published before approval by the U.S. Food and Drug Administration (FDA). But what is often missing is formal, scientific research about how that new treatment compares with existing ones, the authors of the new report discovered.
After assessing studies published between June 2008 and September 2009 in the six most-cited medical journals (Annals of Internal Medicine, Archives of Internal Medicine, the British Medical Journal (BMJ), JAMA, The Lancet and The New England Journal of Medicine), Hochman and his co-author Danny McCormick (of the Cambridge Health Alliance and Harvard Medical School) found a paucity of solid comparative effectiveness studies.
For the sake of clarifying the often-murky field of CER, the two researchers focused only on research that evaluated medications. Of the 328 studies that purported to do this, 104 met the formal definition of CER from the HHS's Federal Coordinating Council for Comparative Effectiveness Research. Among the 104 studies, 43 percent compared two or more medications with each other, 16 percent examined different dosing approaches, and 11 percent compared medications with nonpharmacologic treatments.
"We found that only a minority of medication research focuses on helping doctors use them in an effective, rational way," Hochman says. "The rest of research seems to be devoted to finding novel therapies."
Two percent of the studies evaluated cost effectiveness and 19 percent focused on medication safety, which, notes Hochman, "probably isn't enough."
Hochman and McCormick also found that even those studies that qualified as CER had some "serious shortcomings," Hochman notes. Part of that might be the nebulous nature of CER itself. Although the term CER has been around for years, the Federal Coordinating Council only established the working definition in June 2009, when the papers reviewed for this new study had already been either published or submitted to journals. "It's actually relatively difficult to define studies that are CER—it's sort of a gray area," Conway says, noting that he still thought the new report was "a good overview and estimation of the CER literature."