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- Anthony N. DeMaria, MD, MACC, Editor-in-Chief, Journal of the American College of Cardiology* ()
- ↵*Address correspondence to:
Dr. Anthony N. DeMaria, Editor-in-Chief, Journal of the American College of Cardiology, 3655 Nobel Drive, Suite 630, San Diego, California 92112
On February 17, 2009, President Obama signed into law the American Recovery and Reinvestment Act. Of the $787 billion that was appropriated, $150 billion was allotted for medical issues. Among the most controversial provisions of the medical spending was the allotment of $1.1 billion for comparative effectiveness research (CER). The definition given for CER is the rigorous evaluation of the impact of different options that are available for treating a given medical condition for a particular set of patients. Many see this as a long overdue action that will salvage medicine by providing the data upon which optimal clinical care can be based. Others see it as a dangerous first step toward possible government control of medical practice through reimbursement.
My first reaction upon learning of the CER program was to think, “What's new?” Every day we grapple with choosing the diagnostic and therapeutic modalities that will be most effective for our patients. We pride ourselves on practicing evidence-based medicine. The importance of having data upon which to guide these management decisions has long been well recognized. It seemed like the wheel was being reinvented.
The proponents of CER have marshaled considerable data to support the need for their proposal (1). Their arguments are set in the context of the progressive increase in health care spending, which is currently nearly 16% of the gross domestic product and is projected to increase to 20% by 2016. First and foremost, they point to the marked geographic variation in the application of medical procedures. Using heart disease as an example, Wennberg et al. (2) reported a 4-fold difference in the number of coronary artery bypass graft procedures in Medicare patients in different regions of the country after correcting for appropriate variables. The frequency of bypass grafting was not correlated with the prevalence of myocardial infarction. The variation in medical practice resulted in a nearly 3-fold difference in spending between the lowest and highest spending regions, and expenditures are not consistently related to outcomes (3). Advocates stress that it is unknown which of the different medical practices are superior, and that this is a stark demonstration of the need for comparative effectiveness data.
Given the above issues, several questions occur: if it were easy to obtain such data, why is it that we neither already have them nor are in the process of getting them? In addition, what kind of data would resolve these differences in regional practice? Those proposing CER point out that acquiring approval for a new drug or procedure from the Food and Drug Administration requires only the demonstration of efficacy, not of superiority or even equivalence to alternate modalities. Major supporters of medical research, such as the National Institutes of Health, often do not see comparative effectiveness as within their mission. Although industry provides major sponsorship of clinical research, companies are often reluctant to place their products in a head-to-head comparison with competitors. Thus, advocates maintain that this explains the paucity of CER.
The type of research that would best yield comparative effectiveness data is also uncertain. Obviously, prospective randomized clinical trials, by eliminating or minimizing confounding variables, would provide the highest quality data. However, such trials are very expensive and usually lengthy. In addition, as pointed out in a prior Editor's Page (4), they frequently include only a small percent of the affected population, often excluding patients with comorbidities that are prevalent in clinical practice. Registry data can overcome these limitations, but invariably introduce uncontrolled variables. The same can be said for meta-analysis (5). Thus, it seems apparent that the process of acquiring accurate high-quality comparative effectiveness data will be neither easy nor inexpensive.
It seems to me that the difficulty in acquiring high-quality data is one of the major problems with the CER initiative. The clear cut, black versus white, clinically important differences in effectiveness are often obvious. As has been pointed out, you would not need a large clinical trial to establish the efficacy of parachutes for individuals jumping out of airplanes. Studies for issues in the gray area often yield results showing modest differences that may be subject to interpretation. While large clinical trials yield data applicable to groups of patients, the results must be applied to individual patients with multiple variable characteristics. It is and has always been the responsibility of the physician to apply these results to the individual patients under their care. While CER can provide guidance for clinical decisions, it is unrealistic and would be a mistake to think that one size fits all and that any research could yield data applicable to every individual patient.
The major issue raised by those opposed to or with serious concerns about CER is that it may represent the first step toward an intrusive role for government in the practice of medicine (6). Given the increasing costs of health care, they question whether the major goal of CER will actually be to control expenditures rather than to increase quality. (Parenthetically, it is possible, of course, that CER data could result in increased expenditures if costlier procedures proved more effective or if the manufacturers of drugs/devices shown to be clearly superior increased their prices.) It is clear that for CER to change practice, and thereby possibly reduce costs, the data would have to be accepted and acted upon by physicians and patients. Adherence to the guidance provided by CER could be enhanced by a carrot or a stick, that is, by providing incentives or penalties that could most easily be applied through reimbursement policy. This could result in a powerful role for government in the practice of medicine. For those with the most drastic view, CER could be the first step to the rationing of health care.
My own view about CER is mixed. I certainly agree that the substantial geographic variation demonstrated for virtually all medical services is a bit embarrassing and indicates that we have yet to define the best approach to many clinical conditions. It would be very valuable to have data that defined which approach yields superior effectiveness. Such research has almost certainly not received sufficient attention in the past. Nevertheless, despite the new designation of “Comparative Effectiveness Research,” and the enormous bolus of money directed to the effort, the concept of defining the most effective clinical practices and employing that evidence in our clinical decisions is not new. In fact, it is something that is woven into the fabric of daily clinical practice. Much past and present clinical research addresses comparative efficacy. The Thrombolysis In Myocardial Infarction group alone has performed nearly 40 studies in recent years dealing with clinical effectiveness. I also believe that the proponents may be a bit simplistic about the ease with which they will be able to provide definitive answers with CER. Clinical research is difficult, expensive, and is best at providing answers for groups of patients rather than for individuals. Finally, I think it is undeniable that cost considerations are playing a role in the thrust for CER, and I am a little uncomfortable that this could lead to an interposition of the government between the patient and physician. I do think that much of this can be averted if the studies are done in an open and transparent fashion, involving the traditional research community. Certainly, no one can be against cost-effective medicine, and anything that enhances that goal would be valuable. Although the new emphasis raises questions, on the balance, I think that CER is positive. Additional data on comparative effectiveness should help us make better clinical decisions and result in better care for our patients.
- American College of Cardiology Foundation
- Congressional Budget Office
- Wennberg J.E.,
- Fisher E.S.,
- Skinner J.S.
- Wennberg J.E.,
- Fisher E.S.,
- Skinner J.S.
- DeMaria A.N.
- DeMaria A.N.
- Evans H.