Health Reform Driving Interest in Comparative Effectiveness Research

"We look for medicine to be an orderly field of knowledge and procedure. But it is not. It is an imperfect science, an enterprise of constantly changing knowledge, uncertain information, fallible individuals, and at the same time lives on the line.” ¹
— Atul Gawande, M.D.

It is well understood that patients with similar medical profiles, conditions and symptoms do not always receive the same care. As the noted surgeon and medical writer, Atul Gawande, M.D., has said, variation exists in every aspect of medical practice. That the work physicians do is called “medical practice” underscores the constant state of improvement and, therefore, the very real challenges of variation inherent to treating patients.

Improving health care is a subject at the center of the reform debate. As such, the new programs are emerging to develop a stronger base of empirical evidence to guide medical decision making and justify the use of one course of treatment over the alternatives.

“With our health care system under increasing stress, there is a dire need for more information about the effectiveness of medical interventions, which include not only FDA-approved prescription drugs and medical devices, but many medical and surgical procedures, so that we can make better decisions about the right health care practices for each patient,” according to Cliff Goodman, vice president, The Lewin Group.

One way of addressing care variation is to perform head-to-head comparisons of interventions, Goodman indicated. These comparisons, collectively known as comparative effectiveness research (CER), could help determine which types of care result in the best patient outcomes.

“CER compares the effectiveness, safety, and sometimes other clinical and economic outcomes of two or more health care interventions used to prevent, diagnose or treat diseases, disorders and other health conditions under real-world conditions,” he explained. “CER studies of high-priority health care problems could lead to improved patient outcomes while optimizing use of health care resources. Comparative effectiveness research is a key element of proposals for national health care reform.”

The call to action

The American Recovery and Reinvestment Act of 2009 (ARRA) designates $1.1 billion for comparative effectiveness research, which, according to the Department of Health & Human Services (HHS), “is essential for clinicians and patients to decide on the best treatment. It also enables our nation to improve the health of communities and the performance of the health system.”²

Of the $1.1 billion, the Agency for Healthcare Research and Quality will receive $300 million and the National Institutes of Health and the Office of the Secretary of HHS each will receive $400 million to conduct research assessing the comparative effectiveness of health care treatments and strategies.

The ARRA established the Federal Coordinating Council for Comparative Effectiveness Research (the Council) “to foster optimum coordination of comparative effectiveness research conducted or supported by federal departments and agencies.”³  [Editor’s note: The Lewin Group was awarded a contract by HHS to assist the Council with its strategic framework and inventory to identify gaps and potential priorities for future comparative effectiveness research investment.] In a section entitled “Vision” in its June 30 report to the president and Congress, the Council stated its belief that comparative effectiveness research “is critical to transforming our health care system to deliver higher quality care and more value to all Americans.”⁴

Under the provisions of the ARRA, the Institute of Medicine (IOM) was charged with recommending national priorities for research questions to be addressed by comparative effectiveness research and supported by ARRA funds.⁵ On June 30, the IOM issued its priority list of 100 “direct comparisons of effective interventions, the study of patients in typical day-to-day clinical care.”⁶

“For new drugs and some medical devices, we have sound efficacy data from randomized, controlled clinical trials conducted under largely ideal conditions, typically against a placebo or other inactive comparator, which do establish that these technologies can work, at least versus doing nothing,” Goodman said. “For many medical and surgical procedures, which are not regulated by the FDA, and for other medical devices, we have less comparative evidence. This leaves our health care providers with insufficient evidence for making informed day-to-day care decisions, which also must account for individual patient circumstances, needs and preferences.”

It is worth noting that the topics the IOM selected “go beyond specific treatment comparisons to encompass differences in health care financing, management and delivery,” commented William Crown, Ph.D., president of i3, a division of Ingenix with expertise in clinical trials and study design, drug safety, health economics and outcomes research. “What the government is seeking to learn here is not just what works, but what works better than something else that works,” Crown said. “Eventually it will touch every aspect of the health care continuum.”

Addressing evidence gaps, national variations

To affect health care improvement and fill evidence gaps, the industry needs standard, routine procedures for identifying how effective interventions can be implemented. The IOM stated in its recent report that “incomplete” or “unavailable” information has resulted in more than 50 percent of treatments delivered “without clear evidence of effectiveness.” ⁷ This uncertainty “contributes to a great variability in managing clinical problems, with costs and outcomes differing markedly across the country,” the IOM report stated. Comparative effectiveness research “is a way to identify what works for which patients under what circumstances.”⁸

The disparity between how patients with similar health conditions are treated across the United States also is significant. This variation in the practice of medicine and the use of medical resources has resulted in the overuse, underuse and misuse of care in the U.S.⁹ Researchers at Dartmouth’s Center for the Evaluative Clinical Sciences have found that “variation is not driven by differences in the payment rates across the country but instead by the use of services.”¹⁰

Indeed, there are wide variations among both states and cities; in 2006, Medicare spent $16,351 per beneficiary in Miami and just $8,331 per beneficiary in San Diego.¹¹ Some seek to explain this discrepancy by noting the higher cost of living in Miami. However, in a recent article in The New Yorker, Gawande compared the similar locales of McAllen and El Paso, Texas. The towns share similar size, location and other key characteristics, yet, in 2006, Medicare spent $14,946 per enrollee in McAllen,nearly double the $7,504 it spent in El Paso, even though the quality of care there is no better than in El Paso.¹²

These geographic variations are due in part to a limited amount of high-quality clinical evidence.¹³ “For many conditions, there is a great lack of guidance for physicians around which treatments should be provided,” according to Crown. “To reduce the variance and produce guidelines for physicians, we need to set an agenda that examines how treatments compare to each other.”

Indeed, Crown explained, many biopharmaceutical and medical device companies already are building comparative effectiveness research into their clinical development programs to generate data that will show the value of their products to payers by the time they receive FDA approval. “They know that to get ahead of the curve and get maximum reimbursement, they have to incorporate both the clinical response and the economics associated with their treatments versus another,” he said.

Increasing demand for rigorous CER

In addition to private use of comparative effectiveness research, recent legislative initiatives—from the ARRA to the Medicare Modernization Act of 2003, which funded clinical effectiveness research by the Agency for Healthcare Research and Quality and other entities—demonstrate the growing demand from a wide range of stakeholders for the studies.

According to the IOM report, viable and sustainable national comparative effectiveness research “will require coordination of private and public entities” and, as comparative effectiveness research efforts increase, there will be a commensurate need “for trained experts in biostatistics, epidemiology, systematic reviews, observational and clinical trials, and more refined research methods.”

Closing the evidence gap in comparative effectiveness that exists today will require policy experience, 360-degree health care expertise, access to data and innovative analytical tools, Goodman explained.

To help meet these needs, The Lewin Group announced July 16 that it had established a Center for Comparative Effectiveness Research. The Center combines Lewin’s expertise in the U.S. health care system with key capabilities and assets from Ingenix, The Lewin Group’s parent company.

The new center integrates The Lewin Group’s policy and analytical expertise and government research background; i3 Innovus’ proficiencies in health economics and outcomes research, drug safety, and clinical trials and study design; and the technology tools and capabilities of Ingenix. The Center will offer:

• Development and management of data sets and registries
• Data analysis, including linked data sets and electronic health records
• Tools for analysis of longitudinal health outcomes for treatment effectiveness and drug safety
• Services to conduct and manage institutional review board-approved comparative trials
• CER technical assistance, CER knowledge transfer and consulting on CER methods and policies

“The Lewin Group is superbly positioned to make a considerable contribution to comparative effectiveness research,” said Goodman, who will serve as interim director of The Center until a permanent director is named. “We bring a uniquely qualified set of expertise, data, analytical tools and resources to a high-priority problem in the nation’s health care system.”  

¹ Atul Gawande, M.D.: “Complications: A Surgeon’s Notes on an Imperfect Science” (2002).
² HHS Web site as of July 8, 2009.
³ Federal Coordinating Council for Comparative Effectiveness Research, “Report to the President and the Congress” (June 30, 2009).
⁴ Id.
⁵ Institute of Medicine; “Initial National Priorities for Comparative Effectiveness Research” Report Brief (June 30, 2009).
⁶ Id.
⁷ Id.
⁸ Id.
⁹ Dartmouth Atlas Project Topic Brief, “Effective Care” (Jan. 15, 2007).
¹⁰ Miller, Mark E., Ph.D.; MedPAC “Report to the Congress: Reforming the Delivery System,” Testimony before the Senate Committee on Finance (Sept. 16, 2008).
¹¹ Pear, Robert; “Health Care Spending Disparities Stir a Fight,” (citing Dartmouth Atlas of Health Care) The New York Times (June 9, 2009).
¹² Atul Gawande, MD, “The Cost Conundrum,” The New Yorker (June 1, 2009)
¹³ Tunis, Sean R., MD, MSc; Stryer, Daniel B., MD; Clancy, Carolyn, MD; “Practical Clinical Trials, Increasing the Value of Clinical Research for Decision Making in Clinical and Health Policy,” Journal of the American Medical Association (Sept. 24, 2003).

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