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Denying Care: How Comparative Effectiveness Research Really Works

by Peter J. Pitts
June 01, 2009

If knowledge is power, then what are we to make of various proposals to fund federal research on the comparative effectiveness of different medical treatments? Surely we want those purchasing health care to have the best available information about what works and what doesn’t, right?

The problem is that the U.S. government, through its various health care programs, is also a buyer of health care. Like any buyer, it has a budget. However, the government’s budgeters have a different calculus than do ordinary consumers. Ordinary consumers are open to considering the value offered by new products and adjusting their spending accordingly. Government budget writers, guided by political considerations, are apt to frown on new medical treatments, procedures, drugs, and devices as being too costly—even if they are, in fact, effective.

This conflict is an artifact of the U.S. system of third-party payment for health care. Thus, it should come as no surprise that proposals by President Obama and various members of Congress to expand the role of government in health care come accompanied by proposals to expand comparative effectiveness research. Those proposals, nevertheless, are cause for concern. As Peter Pitts (below) and Helen Evans (page 16) explain in their respective articles, government assessment of health technologies is inevitably a tool for governments to limit their health care expenditures by denying treatments to patients. If knowledge is power, then patients and taxpayers need to understand how comparative effectiveness re-search can be used to serve the government’s budget imperatives rather than their own welfare. —Editor

Today, health technology assessment is a short-term, short-sighted, politically driven policy that results in one-size-fits-all medicine. While it may provide transitory savings in the short-term, current strategies result in a lower quality of care, which results in higher health care costs over time.

Comparing Cost Effectiveness

The United Kingdom’s National Institute for Health and Clinical Excellence (NICE) has issued cost effectiveness decisions for many new medicines. More often than not, after a one- to three-year period of review after a drug has received market approval, the institute has recommended against using the drug because it is not cost effective compared to existing treatments. That is why policy folks in the United Kingdom instead of using “NICE” prefer “NASTY,” for “not available, so treat yourself.”

The drugs the institute has rejected or rationed include Gleevec, a drug that targets a specific pathway that causes stomach cancer. In 2001, Gleevec became frontline therapy for stomach cancer in the United States. To the outrage of most cancer specialists, the institute took three years to decide that it would use Gleevec as a last resort, in a limited dose. It took Gleevec away from patients who have some tumor sites that are responding to treatment and some tumor sites that are not.

The institute has also recommended against paying for Herceptin to treat metastatic breast can-cer, as well as other drugs used to treat osteoporosis, Alzheimer’s, and multiple sclerosis based on comparative effectiveness reviews. Similarly, independent review agencies in Australia, New Zealand, and Canada have done the same.

The institute did not regard any new drugs as clinically less effective then older drugs. Rather, officials judged them to be, relative to their cost, not worth paying for given the additional benefit the drugs provided. The benchmark used in each case was something called a “quality of life year” or a year of life free of disease or infirmity.

Sir Michael Rawlins, chairman of NICE, told the British House of Commons that comparative effectiveness, a means of health technology assessment, is not based on empirical research: “There is no empirical research anywhere in the world, it is really based on the collective judgment of the health economists we have approached across the country,” he said. “It is elusive.”

The problem is that health technology assessment, as it is currently designed, places into conflict the budgeting dilemmas of governments elected for relatively short periods of time with the ever-lengthening life spans of their electorates.

$50,000 Per Year of Life 

In every analysis described, health systems assumed an additional quality of life year was worth about $50,000, the average price of a fully loaded Land Rover. Because most new medicines are targeted therapies that are tested first in critically ill patients, it will be almost impossible to demonstrate significant improvement in well-being or life expectancy for any new medication. The United States Medicare Payment Advisory Commission (MedPAC), the independent federal body established to advise Congress on issues affecting Medicare, never mentions a number, but invokes efficiency enough times to make the point that comparative effectiveness is a tool for controlling costs, not improving the lives of people. Indeed, it notes that “increasing the capacity to examine the comparative effectiveness of health care services” will lead to “[increased] federal administrative spending relative to current law.” That would mean more price controls and government interference in medicine.

It happens that MedPAC and America’s Health Insurance Plans favor large randomized clinical trials to compare older drugs sponsored in part by government agencies and private companies. Randomized trials tend to ignore differences in clinical outcomes due to side effects or genetic variations. So whether you analyze them together or individually, researchers will almost always find no difference in the effect of medicines, a result that is biased in favor of older, cheaper drugs.

Proponents point to two troubling examples. For instance, the Department of Veterans Affairs (VA), along with the National Institute of Mental Health (NIMH), conducted a study comparing older and cheaper drugs for schizophrenia to newer ones. Patients with chronic schizophrenia were assigned to treatment with perphenazine, olanzapine, risperidone, quetiapine, or ziprasidone for up to 18 months.

The first thing NIMH and the VA did was exclude people who froze up and went numb from the older drugs, a common reaction that led to the development of second-generation medicines in the first place. Then the study only compared how well each patient did on each drug. Even then, the researchers assigned their own value to the reasons patients switched, substituting their preferences for those of patients and patient groups. Even though most patients wound up staying on newer drugs for longer periods of time—because of side effects that the study devalued—the researchers claimed that the older drugs were cost-effective.

A Judgment Controversy

Another example of the kind of research proponents of comparative effectiveness swoon over is the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial. That was a five-year, 42,000-patient trial that studied the comparative effectiveness of first-generation blood pres-sure drugs (diuretics) against second-generation medicines in reducing heart attacks. Patients were allowed to switch to a combination of drugs only if they failed on the older medicine first.

The authors of the study concluded that diuretics were cheaper and as effective in reducing death from all forms of heart failure, if not heart attacks, and were therefore cost-effective. But like the trial conclusion in the study that compared drugs to treat schizophrenia, this judgment was not without controversy. As Michael Weber, one of the members of the steering committee overseeing the trial noted, the favorable assessment of the older drugs was driven largely by the fact that African-American patients suffer 40 percent more deaths from stroke because they do not respond well to ACE inhibitors. This fact was well known even before the trial, yet those overseeing the trial still allowed African-American patients to receive a treatment that left them with a higher risk of death.

Even though other research shows that white patients respond at least as well on ACE inhibitors as on diuretics, the VA now has a program where it pays doctors to prescribe blood pressure medications according to guidelines that emerged from the trial.

As currently organized, comparative effectiveness will be used to increase government control over the practice of medicine and expand price controls. It will turn patients into cost centers, not the center of efforts to prevent disease and extend life. Using a combination of cutting-edge in-formation technology and genetic tests, doctors can do a better job than any cost-benefit agency to ensure that their patients get the right treatment at the right time. Many new drugs, including top-selling cancer drugs such as Tarceva and Avastin, have associated genetic tests either on the market or in clinical development that will allow doctors to provide individuals with personalized medicine. New drugs for asthma, depression, HIV, and blood pressure will have similar tests.

Personalized medicine gives doctors and patients control over health care decisions while comparative effectiveness, as it is now defined, will increase government control over the choices doctors and patients make in the future. The battle over the value of medicine and who decides what is valuable will determine who controls health care in America over the next decade.

A health technology assessment model for the 21st century should reflect and measure individual response to treatment based on the combination of genetic, clinical, and demographic factors that indicate what keep people healthy, improve their health, and prevent disease. A rapidly aging society demands a new health care paradigm capable of providing for its needs in the 21st century. Equality of care must be matched with quality of care.

In an era of personalized medicine, one-size-fits-all treatments and reimbursement strategies are dangerously outdated. We are early in this debate, but at least we can all agree that this is not, and must not be, exclusively a debate about saving money. The debate must be about patient care.

Mr. Pitts is President and co-founder of the Center for Medicine in the Public Interest and Senior Vice President, Director for Global Health Affairs for Manning Selvage & Lee. From 2002 to 2004, Mr. Pitts was FDA’s Associate Commissioner for External Relations, serving as senior communications and policy adviser to the Commissioner. This article is excerpted from his article, “‘Comparative Effectiveness’: Government’s Way to Convert Patients into Cost Centers?” published by the Washington Legal Foundation, February 13, 2009, available at www.wlf.org/upload/2-13-09Pitts_LegalBackgrounder.pdf.


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