Health care reform's wild card: the uncertain effectiveness of comparative effectiveness research.

AuthorSaver, Richard S.

Comparative effectiveness research (CER) stands out as the intriguing wild card of health care reform. CER compares competing treatments against each other to determine which interventions work best, supplying critical information for medical decisionmaking and health policy. If CER works as planned, it may be one of the few reform measures in the final health care legislation that could flatten the cost curve while also improving quality. Unfortunately, health care reform has so far failed to bet smart and play the CER wild card effectively. While the Patient Protection and Affordable Care Act invests in CER at record levels and creates an entirely new regulatory framework for oversight of the research, the new law does very little to advance the difficult work of translating CER into actual medical practice. First, CER is costly to conduct and its data often raise more questions than answers. Second, the government's CER agenda seems vague and ill-defined, not consistently focusing on generating research that will help clinicians resolve immediate treatment questions. Third, and most important, physicians likely will remain indifferent to and "tune out" CER. Health law and policy are not setting the right incentives for physicians to adapt their practice patterns to CER and, in some respects, exacerbate the physician-engagement difficulties. The reasons for physician indifference to CER include: lack of financial incentives, suspicions of industry bias in the public/private oversight of the research, threats to clinical autonomy, a commitment to individualized medicine (encouraged by health law, professional ethics, and medical norms) that remains in tension with CER, concerns that CER is a vehicle for crude cost-cutting, and malpractice liability fears. To be truly effective, the new national CER program requires targeted reforms designed to engage physicians more directly with the research. This Article's principal suggestions include greater linkage of CER with reimbursement and liability incentives, enhanced use of academic detailing, and more support for comparative implementation studies that evaluate different strategies for fostering physician uptake of CER.

INTRODUCTION I. BACKGROUND: CER STATUTORY PROVISIONS A. Previous CER Efforts B. The American Recovery and Reinvestment Act of 2009 C. The Patient Protection and Affordable Care Act D. Definitional Issues E. Comparative Effectiveness Versus Cost-Effectiveness F. Limitations on Use of CER II. INFORMATION GAPS AND WHAT DRIVES MEDICAL DECISIONMAKING III. TRANSLATION BARRIERS A. Vagueness and Mission Creep B. Costly Studies That Raise More Questions Than Answers 1. Cost 2. Secondary Data, Less Rigorous Research Methodologies, and Varying Measures of Effectiveness 3. Accounting for Individual Patient Differences 4. Accounting for Individual Provider Differences 5. Keeping Pace with Innovation 6. Uncertain Answers C. Financial Incentives D. Bias and Public/Private Oversight E. Clinical Autonomy and the Technological Imperative F. "Individualized" Medicine G. Liability Concerns IV. RECOMMENDATIONS A. Coverage Rules and Financial Incentives B. Malpractice Liability Incentives C. Academic Detailing D. Comparative Implementation Research CONCLUSION INTRODUCTION

Despite the recent enactment of historic health care reform legislation, (1) serious questions remain whether the entire health care overhaul will implode. Many health policy experts believe that the new law, in primarily focusing on access, does not sufficiently address intractable cost and quality problems in the health care system. (2) The public shares these concerns. (3) As the debate continues, comparative effectiveness research (CER) stands out as the intriguing wild card of health care reform. CER is one of the few reform provisions in the final legislation that, if deployed properly, offers a plausible opportunity to bend the cost curve while also improving the quality of care. Indeed, CER proponents optimistically claim that, over the long haul, CER can radically transform the practice of medicine. (4) Unfortunately, health care reform has so far failed to bet smart and play the CER wild card effectively.

While there are varying and sometimes inconsistent definitions of CER, (5) it involves, at bottom, comparing competing medical treatments against each other to determine which interventions work best. The new governmental push for CER responds to increasing concerns that physicians often make clinical decisions without a solid foundation of credible medical evidence, particularly evidence as to how treatments compare to each other. A recent Institute of Medicine (IOM) report suggests that sound scientific studies support less than half of the treatments that physicians recommend. (6) Uncertainties arising from this information gap contribute to unexpected variations in the treatment of patients with similar conditions and can result in costly, ineffective, and even dangerous medical care. (7)

Health care reform has led to both heavy investment in CER and the creation of a new regulatory framework for oversight of the research. Congress took the first major step with the federal stimulus legislation by including an appropriation of $1.1 billion to fund CER as part of the American Recovery and Reinvestment Act of 2009 (the Recovery Act). (8) The recently enacted health care reform law, the Patient Protection and Affordable Care Act (PPACA), (9) provides an additional stream of CER funding--up to $500 million per year by 2013 or 2014---and establishes a new oversight entity, the Patient-Centered Outcomes Research Institute (the PCOR Institute), to direct the nation's first comprehensive CER program. (10)

CER attracts considerable enthusiasm as a tool for health care reform because it differs from conventional medical research in important ways. Traditional studies ordinarily evaluate an experimental intervention's general effectiveness by comparing it to a placebo. CER instead focuses on how effective treatments are relative to each other. In other words, traditional research typically asks, "Does this work?" whereas CER asks the question practicing physicians really want to know: "Is this better than that? (11) Also, conventional medical investigations usually evaluate new technology under tightly controlled, highly artificial conditions that exclude many subjects, whereas CER includes treatments already adopted in clinical practice and studies populations more representative of typical patients. (12) As such, CER promises to develop better information for medical decisionmaking in real world set, tings. In theory, better information will translate to better medical care. Various commentators predict that CER has the power "to reshape major portions of the practice of medicine," (13) that it provides "the scientific scaffolding for ... revolution" (14) in medical practice, and that it ushers health care into a new "era of comparative effectiveness." (15)

Rather than revolutionizing medicine, however, this Article cautions that the emerging era of comparative effectiveness is in danger of becoming disappointingly ineffective. Serious legal and policy obstacles jeopardize the health care system's ability to make productive use of governmentally funded CER.

First, CER already has considerable political baggage after being swept into the larger, acrimonious battles over health care reform. Opponents of the Democrats' health care proposals pointedly focused on CER, charging that it would lead to rationing of health care, (16) governmental interference in the doctor-patient relationship, (17) and, more ominously, the empowerment of "death panels." (18) Also, stakeholders waged hard-fought turf battles over how to administer the new national CER program. The new reform law jettisons the federal-commission approach of previous legislation (19) in favor of ceding direct authority to private interests through creation of the PCOR Institute, a nonprofit corporation that will include drug, device, and insurance company representatives on its governing board. (20) CER faces longer-term political risks as well. Various physician groups, drug companies, and device manufacturers understandably view CER as a threat because the research may question the necessity and value of their products and services. These powerful stakeholders can be expected to use the political process to discredit certain studies and to weaken governmental support for CER more generally, as has happened in the past. (21)

While not minimizing these obstacles, this Article contends that health care reform's new push for CER faces a more fundamental problem: lawmakers have essentially deranged CER, deploying it under conditions that will leave it underpowered. In part to appease critics who fear that CER will lead to rationing and crude cost control, a great deal of legislative and political attention has been devoted to restricting its use. (22) This leaves unresolved the critical question--what will be done with the information?

Not much. Many physicians seem unlikely to change clinical practice patterns, notwithstanding the outcomes of CER studies. Health law and policy are not setting the right incentives for physicians to incorporate CER into regular clinical practice and, in some respects, exacerbate the physician-engagement difficulties. This Article explores why. It also considers how health law and policy tools could better support CER's translation into medical practice.

A key assumption of this Article is that the success of the government's new CER program depends most on targeted physician engagement. The intended audience for a national CER program certainly includes other stakeholders. As CER proponents maintain, comparative effectiveness evidence should empower patients and payers as consumers of health care, equipping them with better information to navigate the complicated health care system. (23) But...

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