At Capitol Hill Briefing, Experts Weigh Benefits, Impact of Comparative Effectiveness Research

Comparative effectiveness research, the use of science to assess the value of medical treatments, has become part of the debate over health care reform but remains widely misunderstood, according to experts at a recent Capitol Hill luncheon briefing organized by AAAS and The Hastings Center.

Steve Findlay
Steve Findlay

The research draws on many sources of data, including traditional controlled clinical trials as well as patient registries, observational studies and medical records. The intent is to compare similar treatments, such as drugs, or very different approaches such as surgery versus drug therapy, and weigh the benefits and risks of each approach. A frequent issue is which types of patients would benefit most from a particular treatment.

Comparative effectiveness research, or CER, also can grapple with broader questions such as the impact of health policies on the delivery of care, and some analysts increasingly see it as a way to help control spiraling medical costs.

But Steve Findlay, senior health policy analyst for Consumers Union, told the 15 December briefing that comparative effectiveness research is not about rationing of health care. “It’s not primarily about saving money,” he said. “And it’s certainly not about limiting a doctor’s judgment.” Rather, Findlay said, CER is aimed at putting more and better information into the hands of doctors and patients that is directly relevant to comparing treatments and making decisions about medical care.

Advocates of comparative effectiveness research do need to do a better job explaining what it is and is not, Findlay said. Even before the advent of CER, he said, consumers often were confused by medical research studies and how inconclusive they often seem.

Interest in CER is driven, in fact, by the disconcerting lack of solid evidence regarding the effectiveness of many medical treatments. A recent report from the U.S. Institute of Medicine estimated that fewer than half of medical interventions used today are supported by clear evidence of effectiveness. “This uncertainty contributes to great variability in managing clinical problems, with costs and outcomes differing markedly across the country,” the report said.

Provisions for CER are included in both the House and Senate health care reform bills. The American Recovery and Reinvestment Act of 2009, the economic stimulus package, also provided $1.1 billion to fund research on the effectiveness of different medical treatments for specific conditions.

Dr. Steven Pearson
Dr. Steven Pearson

Dr. Steven Pearson, president of the Institute for Clinical and Economic Review, told the briefing that CER synthesizes existing medical data but also is meant to generate new evidence that can be used to highlight the clinical approaches likely to provide the most benefit to patients.

But even when findings are agreed upon by an expert review panel, the outcome of comparative effectiveness research can be controversial, as was the case in the recent flap over mammography guidelines issued by a federal advisory task force. The task force recommended that women not begin routine mammography screenings until age 50 and then receive them once every two years. That is a shift from guidelines issued in 2002 that called for mammograms every one to two years for all women older than 40.

The new recommendations caused an uproar, with patient advocacy groups, many physicians, and members of Congress expressing disagreement with them. U.S. Health and Human Services Secretary Kathleen Sebelius distanced her department from the recommendations, advising patients to “keep doing what you have been doing for years—talk to your doctor about your individual history, ask questions, and make the decision that is right for you.”

Pearson, who is also a senior fellow at America’s Health Insurance Plans and a visiting scientist in the Department of Bioethics at the U.S. National Institutes of Health, said that any effort to use CER results for medical guidance should include clinician and patient organizations in leadership roles from the outset. He called for a formal process that rapidly integrates new research data into professional guidelines and criteria on appropriate treatments.

Dr. Richard Payne
Dr. Richard Payne

But Dr. Richard Payne, professor of medicine and divinity at Duke University and director of Duke’s Institute on Care at the End of Life, said there is an art to medical practice that always will extend beyond hard numbers and science. He sketched some of the dilemmas through his personal experience in arranging care for a 102-year-old aunt who has moderately severe Alzheimer’s disease.

Six years ago, Payne’s aunt was rushed to the emergency room with an inflamed gall bladder. Should surgeons proceed with removal of the elderly woman’s gall bladder? There was little evidence in the medical literature on her chances for survival or a return to a relatively normal life (she lives in a family-style rest home with several other cognitively impaired women). “I try to practice evidence-based medicine,” Payne said. “What should we do?” The family, faced with scarce medical evidence but an abiding concern for a loved one, decided to approve the surgery. The aunt recovered and left the hospital about a week later.

Payne questioned whether comparative effectiveness research could ever be helpful in guiding such individual decisions. While he dismisses alarmist concerns that health care reform might bring “death panels” with the power to dictate care decisions at the end of life, Payne argues that strict adherence to rigid protocols drawn from evidence-based treatment approaches will never provide all the answers.

“There will never be the kind of data that will allow us to make fully evidence-based decisions for individual patients,” Payne said. “Medical care is ultimately practiced at the level of individual patients.”

Alan I. Leshner, the chief executive officer of AAAS and former head of the National Institute on Drug Abuse, said that CER “will be a framework, not a script, for conversations between doctors and patients.” But Leshner, who moderated the panel discussion, said the value of effectiveness research is clear.

Over the years, Leshner said, he has been “at times appalled and frightened” by the lack of scientific evidence surrounding some medical interventions. At the same time, he said, proponents of CER must do a better job of marketing the value of rigorous scientific investigation for the nation’s health care.

Pearson said that CER is “not about trying to smudge over all the differences between individual patients and populations. This is about finding those [differences] with the new research and then clarifying them” through the assessment process.

The evidence developed can help foster more prudent use of costly medical interventions. Insurance companies should not use the data simply to make “cover or no cover” decisions, Pearson said. They can also use the information in more innovative ways, he said, including payment for coverage as long as a procedure is done as part of a clinical trial or payment based on demonstrated effectiveness of a procedure compared to existing interventions.

Pearson warned that medical costs are rising so rapidly that the United States may be in a “health care bubble” that is about to burst unless there is more attention paid to what works and what does not.

With many people confusing “comparative effectiveness” with “cost effectiveness” and expressing concern about rationing of health care, Findlay said, advocates of CER should not duck the money issue. “It’s time to play offense on this issue, not defense,” he argues. “CER may in the long run save money by preventing unnecessary treatment and poor care. That is a good thing.” In some areas of clinical practice, he said, CER also is likely to increase costs as the effectiveness of a particular medical intervention is convincingly demonstrated.

It is important not to demonize CER, Findlay said, and to keep in mind why some industry groups are fighting provisions in health care reform bills that would expand use of the technique. Some drugs and devices may lose out, he said, as more evidence is gained on their relative effectiveness.

“There is uncertainty and ambiguity with all of this stuff,” Findlay said. “Consumers continue to be persistently confused about it. We’re going to have to work very hard, I think, to build tolerance for that ambiguity. We’re going to have to work as hard as we can to explain CER better.” The payoff, if it all works properly, could be the prevention of unnecessary risk and a strengthened doctor-patient relationship, he said.

Pearson, too, said that comparative effectiveness research, done right, could have a broad impact on medical care in the United States. “This is a tool that we need to use if we are to have any hope of advancing the greater interest, not only of our country, but of individual patients and doctors,” he said.