Doctors say it is often difficult in the course of day-to-day practice to know how various drugs and other medical treatments stack up against one another or which are best to prescribe for their patients.
There’s plenty of research on the efficacy and safety of drugs, medical devices and other treatments.
But doctors say that information typically isn’t organized in a useful way or generally is most available from the vendors.
Often, they are unable to draw on unbiased information comparing the effectiveness of competing drugs or analyze well the different treatment approaches, such as surgery versus drug therapy, and compare the relative costs.
Federal policymakers say a potential solution to these problems is greater use of comparative effectiveness research, which the Congressional Budget Office has defined as “a rigorous evaluation of the impact of different options that are available for treating a given medical condition for a particular set of patients.”
In a 2007 report, the CBO offered this example: A recent trial found that older, relatively inexpensive drugs for treating high blood pressure, known as diuretics, were more effective in preventing cardiovascular disease in patients age 55 or older than commonly used newer drugs. That kind of head-to-head study isn’t normally conducted when a new drug is vetted for safety by the federal Food and Drug Administration.
Without hard evidence, according to the CBO, “decisions about what treatments to use often depend on anecdotal evidence, conjecture, and the experience and judgment of the individual physicians involved. In many cases, that basis may be sufficient… But if the benefits of a treatment — or risks of not providing it — are less obvious, the lack of hard data makes determining the appropriate choice of treatment difficult.”
Comparative effectiveness research is not a new concept. Private-sector companies have accumulated evidence through clinical trials and technology assessments. And private health insurance companies use comparative effectiveness research from claims data to help decide which treatments they cover.
But some experts believe that less than half of all medical care is based on adequate evidence of its effectiveness.
Supporters say if there were more comparative effectiveness research and if the results of it were made widely available, health costs could be lowered. For example, the research could show when older, less expensive drugs are better for patients than newer more expensive treatments being promoted by drug companies and other vendors.
One reason the research has not been more widespread is because it is hard to find populations that can be compared in studies, said Dennis Cotter, president of Medical Technology and Practice Patterns Institute, Inc., a nonprofit research institute in Bethesda, Md. that studies new medical technologies.
“It can be done — there was a Canadian study on an endoscopic approach to treating knee problems compared to conservative medicine,” he said. “It found that the conservative medicine did better for patients. But were the patients really identical? It’s real hard to do, because patients react differently to different interventions.”
The federal government has at various times had several programs to encourage more comparative effectiveness research, including creation of the Office of Technology Assessment, meant to aid Congress in using scientific evidence to shape policy.
The National Center for Health Care Technology, a short-lived program in the late 1970s and early 1980s, was also created to look at the usefulness and efficiency of new and established medical technologies.
The federal Agency for Healthcare Research and Quality now has some evidence-based medical research responsibilities — and has received $300 million from the American Recovery and Reinvestment Act to increase its comparative effectiveness research.
Cotter served as the deputy associate director for Medical and Scientific Evaluation at the National Center for Health Care Technology, which was the first organized effort to compare the various facets of medical technology.
It will take leadership, if the research is to become useful and commonplace, he said.
He cited the example of a popular anemia drug that is widely used and well-reimbursed by Medicare. His organization found it had not been compared to other similar drugs, even though Medicare spent about $2 billion on the drug in 2004. Cotter said dosing levels have increased since the drug was released, though few studies exist to prove that strategy is effective.
“There is no office to do this,” he said.
The Centers for Medicare and Medicaid Services, the Food and Drug Administration, the AHRQ, Congress and the newly-created White House Office of Health Reform could all take part in conducting and then relying on evidence gleaned from comparative effectiveness research.
“This is the kind of stuff that has never been reported before now—– these techniques were not around 20 years ago,” Cotter said. “These studies directly impinge on policies. The challenge now, is once we do the studies, to get it in to the literature. The next step is implementation.”
It’s hard to estimate how much is now spent on comparative effectiveness research, according to the CBO. A 2007 report from the Institute of Medicine made a rough estimate that $2 billion is spent each year on all comparative effectiveness research in the private and public sectors.
About $1.1 billion was included in the American Reinvestment and Recovery Act to further the research. The funds will be divided between government agencies such as the National Institutes of Health, the Department of Health and Human Services, and the Agency for Healthcare Research and Quality.
“Getting to the point at which additional research on comparative effectiveness could have a noticeable impact on health care spending would take several years,” the CBO said. “It would probably be a decade or more before new research on comparative effectiveness had the potential to reduce health care spending in a substantial way.”
-Sarah Green is a staff writer for KHI News Service, which specializes in coverage of health issues facing Kansans. She can be reached at sgreen@khi.org or at 785-233-5443, ext. 118.