The Perils of Government-controlled Comparative Effectiveness

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As government-controlled Comparative Effectiveness Research (CER) seems soon to become a fait accompli in the US, I recall the words of the great Edmund Burke (1729–1797), the British statesman and philosopher generally considered to be the philosophical founder of modern political conservatism, to remind our legislative powers not to ignore the history and evolution of Great Britain’s National Institute for Health and Clinical Excellence (NICE): “Those who don’t know history are destined to repeat it.”

The British officials who established NICE in the late 1990s said that it was created so that the National Health Service (NHS) would use ‘best practices’ in medicine. In 1998 the Guardian reported: “Health ministers are setting up NICE, designed to ensure that every treatment, operation, or medicine used, is proven best. It will root out under-performing doctors and useless treatments, spreading best practices everywhere.” However, as health costs exploded, NICE became the heavy that reduced spending by limiting treatments that Britons are allowed to receive from the NHS. In the process, NICE became a rationing board—an eventuality not anticipated by its founders. Examples of this rationing are both legion and painful to recount: NICE denied the use of lapatinib and Sutent, which prolong life in certain types of breast and stomach cancer; Macugen, used for patients with macular degeneration, was denied outright; and Lucentis, effective in a specific type of macular degeneration, was approved in only about one in five patients with the disease, and even then only for use in one eye, relegating the other to blindness.

Andrew Dillon, when Chief Executive Officer of NICE, observed that “when treatments are very expensive, we have to use them where they give the most benefits to patients.” This draconian therapeutic philosophy appears to be pervasive in NICE. For example, Aricept is limited in the early stages of Alzheimer’s disease, even though UK doctors contended vigorously that the most effective way to slow the progress of the disease is to use the drug at the first sign of dementia. NICE countered that this medication was not ‘cost-effective’ in the early stages of Alzheimer’s disease. Similar restrictions by NICE in the exhibition of useful medications apply, for example, to rheumatoid arthritis, multiple sclerosis, and multiple myeloma. Private insurers in the US often cover all or at least part of many prescriptions denied by NICE. Rationing by NICE also extends to a spectrum of surgical procedures and diagnostic modalities.

Nevertheless, the various appraisals of NICE are complex and not uniformly negative. The Hastings Center in the US published a critique titled “Comparative Effectiveness Research (CER) and Rationing: Lessons from NICE.” I had rather expected that this report would include a discussion of rationing practiced by NICE, of which those soon to be in charge of CER in the US should be aware. Although noting a Wall Street Journal editorial stating that “The CER outfit will start to ration care to control costs,” and quoting George Will that “CER would dramatically advance government control—and rationing—of healthcare,” the Hastings report countered that: “To support their ‘slippery slope’ argument, the critics point to Britain’s NICE, which uses both CER and cost-effectiveness analysis to advise the British National Health Service (BHS) what should be provided through the service.”

The Hastings report went on to say categorically that the critics of NICE are wrong. I presume that this position is taken in response to NICE’s critics, who were correct in pointing out that NICE uses not only CER but also cost-effectiveness in authorizing NHS services. Inevitably, cost-effectiveness is a matter of judgment, and therefore in effect becomes a codeword for rationing. This is in keeping with what Andrew Dillon had already noted above, that “when treatments are very expensive, we have to use them where they give the most benefits to patients.” Note that Dillon’s emphasis is on ‘patients,’ meaning society in general, not on the individual patient. This brings us to the growing recognition that in our society, doctors, NICE, and the soon-to-come government-run CER in the US are constrained by the exigencies of overwhelming demand and finite resources, in both the private and the governmental sector. Thus, medicine’s ancient tradition dating back to Hippocrates, that the physician’s ultimate obligation is to serve the interest of his or her patient, is being abrogated and replaced by a new philosophy wherein the physician has a dual responsibility to both patient and society. This will inevitably dilute, temper, and diminish the doctor’s commitment to the individual patient. Ironically, this new philosophy has been endorsed in a paper published simultaneously in the current issues of The Annals of Internal Medicine and The Lancet, titled ‘Medical Professionalism in the New Millennium: A Physician Charter.’ This manuscript is a project of the American Board of Internal Medicine Foundation, the American College of Physicians–American Society of Internal Medicine Foundation, and the European Federation of Internal Medicine.

In December 2000, Richard Smith, Editor of the British Medical Journal (BMJ), published an editorial titled ‘The Failings of NICE.’ He proceeded without equivocation to say that: “One failing of NICE is that it is living a double lie. The first lie, which is as Orwellian as its name, is for NICE to deny that it’s about rationing healthcare, which might be defined as denying effective interventions. Denying ineffective interventions is not rationing; rather, it’s what the Americans call a no-brainer.” Smith goes on to say that the second lie is to imply that if the evidence supports an intervention, it is offered, and without the evidence it is not offered. He denies that it is so simple, that these decisions can be made with some data and a computer. He contends, rather, that this lie corrupts the concept of evidence-based medicine, long championed by the BMJ.

Richard Smith then expresses what I believe to be a profound philosophy: “The evidence supports decision-making, but the evidence can’t make the decision. The values of the patient or the community must be part of the decision. Effective interventions have adverse effects. How can benefits be weighed against risks? … Similarly, treatments that are highly cost-effective in those at high risk are also effective in those at low risk, but at very high cost. Deciding where cost-effectiveness ends is not a technical, but an ethical judgment.”

Smith believes that another failure of NICE is that it is concerned primarily with technologies that are new and expensive. He believes that a better approach would be to consider all interventions. Otherwise, a system preoccupied with new technologies will inevitably lead to unintended waiting lists and delays, amounting to discrimination and ultimately to rationing among the elderly and the mentally ill. Smith closes with the admonition that perhaps NICE had to exist to lead the way to something better. He believes that Britain could benefit from an institution that admits that it is about rationing, is ethical and transparent in its judgments, distances itself from politicians and the pharmaceutical industry, and is directly responsible to the public. One can only praise and admire Smith’s utopian vision of what NICE might have been and what his mythical replacement might someday become, for which he has already proposed a name: ‘The Committee for Honest and Open Rationing’ (CHOR).

Having heeded the injunction of Edmund Burke by becoming cognizant of the origins, evolution, and history of NICE, since it may become a model for CER, it seems appropriate to consider the prospects and perils of CER in the US. Ironically, while writing these words, I have just learned that President Obama has abandoned the term ‘comparative effectiveness research’ in favor of ‘patient-centered health research.’ This is reminiscent of his invoking ‘overseas contingency operations’ to replace ‘war on terror.’ Nevertheless, I shall continue to use CER in this manuscript.

The economic stimulus bill, The American Recovery and Investment Act (ARRA), includes $1.1 billion for CER without providing a definition of what that entity is. However, CER is defined in the Agency for Healthcare Research and Quality (AHRQ) bill as follows: “A type of healthcare research that compares one approach for managing a disease to the results of other approaches. CER usually compares two or more forms of treatments, such as different drugs for the same disease. CER can also compare types of surgery or other kinds of medical procedures and tests. The results are often summarized in a systematic review.” CER will be administered by a Federal Coordinating Council (FCC) of 15 federal employees, half of whom must be physicians or other experts with clinical expertise. Some have suggested that the FCC is the first step toward an organization modeled after NICE in the UK, which is why this editorial has devoted so much attention to NICE, leading to the conclusion that emulating NICE is an eventuality, deferring to the wisdom of Shakespeare, “to be honored more in the breach than in the observance.”

The budget for CER is currently proposed to be allocated as follows: $400 million to the discretion of the Secretary of the Department of Health and Human Services (HHS), $1.5 million to the Institute of Medicine for a report on where to focus the attention of CER initially, $400 million to the Office of the Director of the National Institutes of Health (NIH), and $300 million to AHRQ.

Ultimately, the success of CER will depend on the performance of the FCC and of those to whom the tasks of executing the CER are assigned. I cannot help but feel, however, that the administration is somewhat naïve in underestimating the complexity of the CER that it has proposed and in thinking that the medical profession is ‘flying blind’ without knowing ‘what works and what does not.’ It seems unaware of the penetrating debates on the pros and cons of medications, procedures, and devices that go on at the meetings of the American Colleges of Cardiology and Surgery and the American Heart Association, and in their journals, as in dozens of other specialty societies. Furthermore, all aspects of medicine and surgery are constantly being tested in the crucible of experience. Sometimes it takes years to unravel with certainty the optimal approaches in medicine and surgery. These observations are in no way intended to denigrate CER, but rather to suggest that it may be a embarking on a perilous journey.