Perspective
Health Care Reform and the Need for Comparative-Effectiveness Research
N Engl J Med 2010; 362:e6January 21, 2010
- Article
Health care reform will eventually pit the goal of expanding health insurance coverage against strong pressure to reduce the growth in health care costs. If left to measures in the proposed reform legislation, cost containment will be driven primarily by marketplace incentives, programmatic initiatives, and organizational changes that would partially offset the costs of expanding coverage.1 These proposed economic policy changes and incentives, however, are not guaranteed to lead either to individual clinical decisions that will be in patients' best interests or to enhancement of the public's health. Rather, these forces are relatively blunt instruments that might nudge the health care system toward efficiency, without precise direction.
Thus, to ensure that the health of the population is enhanced, rather than diminished, by the reforms, these measures must be accompanied by the sharper tools of comparative-effectiveness research (CER). Unless we are willing to allow our health care to be driven solely by financial and regulatory incentives, CER should become an important part of the equation. Rather than being resisted by the clinical and public health communities, it should be embraced.
CER is included in one way or another in both of the current health care reform bills. The Senate bill calls for the establishment of a semi-independent commission to sponsor this type of research, whereas the House bill would place CER under the auspices of the Agency for Healthcare Research and Quality (AHRQ). Both bills propose modest new governmental funding for this type of research.
Virtually every Western country that has reformed its health care delivery system has instituted CER in one form or another to prevent blunt incentives from inadvertently harming patients and to foster intelligent changes that can improve both the efficiency and the quality of care.2 Unfortunately, there is still a widespread lack of understanding about what CER will do — and fear that it will do more harm than good, in part by threatening individual physicians' autonomy and professionalism. And though both bills specifically prohibit the use of comparative-effectiveness information in making coverage decisions, there are fears that patients will be denied effective care on the basis of CER's findings.
This view is shortsighted and perhaps even misdirected, since changes in coverage and care delivery must in fact be guided by knowledge about what is valuable to preserve in our health care system. The findings from CER will provide a buffer against “blind” cost containment. For example, for certain categories of patients who have had a myocardial infarction accompanied by nonsustained ventricular tachycardia, expensive implantable defibrillators have been shown to be more effective than medications and to be an efficient use of resources in terms of the cost per life-year gained.3 This finding does not mean that dollars are saved, but rather that such defibrillator implantation is a good buy as compared with alternative uses of funds — so that overall health will be enhanced if resources are allocated to defibrillator implantation for appropriate patients rather than to some other use that costs the same but achieves less health improvement. Such information from CER should be used to make the case against restricting the use of implantable defibrillators in this group of patients.
As potentially critical as CER may be as a tool for accomplishing intelligent cost containment by preventing denials of coverage that would have adverse effects, it could have an even greater effect by identifying preferred therapies. Insofar as it is a sharp instrument, CER can inform and promote changes in care and outcomes that would never have been envisioned, much less achieved, through shifts in the financing, organization, or delivery of care. Keener insights are needed if we are to devise ways to improve and maximize health with limited resources.
In this era of high-tech care, there is little likelihood that less-complex and cheaper therapies will emerge as preferred approaches, unless CER is allowed to lead to the identification and validation of such treatments. For example, inexpensive diuretic medications have been shown by the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial (ALLHAT) to be more effective than more-expensive treatments such as angiotensin-converting–enzyme inhibitors, calcium-channel blockers, and alpha-blockers.4 We need such research to suggest ways in which the proposed new financial incentives can be applied effectively and safely.
In addition to having direct effects on clinical decisions, CER should also improve medicine in other ways. Changing the climate so that health care decision makers favor data from CER will create disincentives for the development of “me-too” drugs and devices and render CER a catalyst for effective innovation. It should then become clear to pharmaceutical companies and device manufacturers that a new product based solely on advances identified only in laboratory or animal studies will not be accepted in the medical marketplace unless it is also proven to be superior to currently available therapies or diagnostic devices. Once the medical community begins to expect and demand clear evidence of superiority, manufacturers will have to devote more of their resources to the discovery of true advances. Furthermore, if the emphasis is also on cost reduction, there is an added incentive to do the research and development that will bring to the marketplace not only superior therapeutics, but also equivalent interventions that are cheaper than their predecessors and therefore more cost-effective. Clement and colleagues5 recently described the process of introducing innovative drugs into three countries (Britain, Australia, and Canada) that already use comparative-effectiveness findings. Only drugs with strong evidence were approved on first submission. For example, all these countries rapidly approved ranibizumab for age-related macular degeneration because of the strong supporting evidence of its superiority to alternatives. On the other hand, an application for marketing approval of insulin glargine, which has only slight advantages over neutral protamine Hagedorn (NPH) insulin, was resubmitted five times in Australia before it was approved at a much lower price than had originally been proposed, and Britain's National Institute for Health and Clinical Excellence approved the drug only for a subgroup of patients: those with type 1 diabetes and a small subgroup of those with type 2 diabetes who might be more likely to benefit. At present, incentives for such decisions are absent in the United States.
Finally, CER will be a stimulus for the academic medical and public health communities to develop a research agenda that is responsive to the needs of the clinical community, providing health care professionals with information for clinical decision making. At the same time, the push for CER will result in greater demand and thus more opportunities for physician-scientists to do this type of research.
The medical and public health communities should be enthusiastic about CER and support it. It is physicians' first line of defense against blind cost containment. On the basis of CER, our drug and device industries will be encouraged to develop products that really matter. We will have a medical research establishment that generates the information we need and that requires and produces opportunities for physicians to engage in and lead such research. Important medical decisions should be guided by the scientific community, not the capricious nature of the marketplace.
Financial and other disclosures provided by the authors are available with the full text of this article at NEJM.org.
From the Department of Public Health, Weill Cornell Medical College, New York. This article (10.1056/NEJMp0912651) was published on January 6, 2010, at NEJM.org.
- References
References
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Davis K, Guterman S, Collins SR, Stremikis K, Rustgi S, Nuzum R. Starting on the path to a high performance health system: analysis of health system reform provisions of reform bills in the House of Representatives and Senate. New York: The Commonwealth Fund, November 2009.
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Mushlin AI ,Hall WJ ,Zwanziger J , et al. The cost-effectiveness of automatic implantable cardiac defibrillators: results from MADIT. Circulation 1998;97:2129-2135
Web of Science | Medline4
Pollack A. The evidence gap: the minimal impact of a big hypertension study. New York Times. November 28, 2008:B1.
5
Clement FM ,Harris A ,Li JJ ,Yong K ,Lee KM ,Manns BJ . Using effectiveness and cost-effectiveness to make drug coverage decisions: a comparison of Britain, Australia, and Canada. JAMA 2009;302:1437-1443
CrossRef | Web of Science | Medline
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