AAMC Reporter: September 2009

Overview: Comparative Effectiveness and Health Care Reform

What is Comparative Effectiveness Research (CER)?

In a nutshell, CER attempts to determine what therapeutic approaches work best for what patients under what circumstances. CER goes far beyond simply comparing two different drugs, though that is how it is sometimes described.

CER studies investigate the effectiveness of different approaches to diagnosing, preventing, and managing diseases, as well as attempting to determine which patients will benefit most from different surgeries, medical devices, and drug therapies. In companyfunded clinical trials, the efficacy of prospective drugs or devices is evaluated by comparing them to placebos or no treatment, often under highly controlled conditions. Treatments are usually not compared with competing drugs or products in typical clinical settings.

Who pays for CER?

Overall, clinical research is funded by private industry and the National Institutes of Health (NIH). In general, however, the respective goals of each are to bring new products to market and advance scientific research— not determine the best treatment or therapy in typical practice. Private industry may be reluctant to invest heavily in CER, mainly because each trial would have a "winner" and a "loser" that could result in lost market share for a specific product or company. That is why, as of now, the government is taking the lead on CER. The American Recovery and Reinvestment Act (ARRA) set aside $1.1 billion for CER in the next decade. The Agency for Healthcare Research and Quality (AHRQ) and the National Institutes of Health (NIH) will receive $700 million for CER, and the Department of Health and Human Services (HHS) will receive $400 million to accelerate the development and dissemination of CER data.

A June 29 report from the newly formed Federal Coordinating Council for Comparative Effectiveness Research (FCCCER) recommended that the $400 million in CER funds destined for HHS be primarily invested in data infrastructure, to be disseminated by "linking current data sources to enable answering CER questions," developing "distributed electronic data networks and patient registries," and enabling private sector partnerships.

I have heard that CER is controversial. Is this true?

Very much so. CER advocacy has made for strange political bedfellows. Consumer groups, labor unions, and medical researchers have found themselves on the same side as large employers, private insurers, and pharmacy benefit managers, for whom CER is likely to help rein in costs.

However, CER carries high stakes for private industries, which stand to lose money if a CER trial renders a certain product less effective than another. Many interest groups have lobbied furiously against CER, and often claim that the findings will be used to deny coverage for more expensive treatments. Republican lawmakers and some voters have voiced concern that CER will allow the government to make personal patient decisions by enforcing across-the-board clinical guidelines and protocols and, perhaps, ultimately deny access to certain treatments.

CER advocates counter that scientists and research agencies will be in a much better position than corporations to conduct the research that gives patients and physicians the information they need. They point out that this kind of research information is a public good just like the basic research funded by the NIH. Furthermore, all proposed CER legislation makes it clear that CER should inform patients, not determine insurance coverage policy.

What therapeutic alternatives will researchers first evaluate?

In the ARRA, Congress asked the Institute of Medicine (IOM) and the FCCCER to recommend to AHRQ, NIH, and HHS how best to invest these stimulus funds. On June 30, the IOM submitted to Congress a prioritized list of studies for national CER. To come up with this list, the IOM solicited the feedback of multiple stakeholders and more than 1,700 questionnaire respondents. From that feedback, the IOM came up with 100 CER topics that it believes will yield the biggest advances.

The IOM's 100 recommended research priorities range from studies on behavioral therapies to disease management to straightforward drug comparisons, and the topics covered range from osteoporosis to acupuncture. The IOM also urged the government to subsidize the education of researchers skilled in conducting CER.

What methodologies will inform CER?

The IOM report recommends that head-to-head randomized control trials be used to evaluate about half of the 100 research priorities it identified. It recommends prospective observational studies, database reviews, and systematic literature reviews to study the best way to treat the remaining priorities.

What are some examples of CER programs and initiatives that are in development or already in place?

Despite limited national investments in CER, this work is already undertaken in a variety of academic institutions. AHRQ funds 41 CER centers nationwide, and the NIH has funded a variety of high-profile CERtype clinical trials over the years. The NIH also funds the Clinical and Translational Science Awards program, which now supports infrastructure and training in clinical research (including CER) in 46 states. (Some institutions receive CER support from both agencies.)

There are a variety of other CER activities as well. The Drug Effectiveness Review Project at Oregon Health and Science University details its findings on competing drugs in various consumer-oriented publications, and is used by 14 states to help purchasers like Medicaid lower their costs. The center's scientists have faced stiff opposition from drug companies and some patient groups.

What does the future hold?

The Obama administration has clearly made a large investment in CER that will continue for several years. As the health care reform debate accelerates in Washington, CER stands to be a major point of both enthusiasm and contention among lawmakers. Various reform bills and influential politicians have expressed competing visions for CER, ensuring that challenging discussions are likely to continue.