American Journal of Law & Medicine

Comparative Effectiveness Research under the Patient Protection and Affordable Care Act: Can New Bottles Accommodate Old Wine?

CONTENTS  I.   INTRODUCTION II.  THE ROAD TO COMPARATIVE EFFECTIVENESS RESEARCH      A. Early Efforts at Technology Assessment      B. The Ascendency of Health Services Research      C. The Saga of Medicare Coverage Policy      D. The AHRQ, the NIH, and Comparative Effectiveness         Research         1. Federal Efforts in Comparative Effectiveness Research            Under the American Recovery and Reinvestment            Act of 2009         2. The Brouhaha over Comparative Effectiveness Research            in 2009 III. THE PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE      A. Key Definitions      B. Purpose of the Institute      C. Duties         1. Identifying Research Priorities and Establishing a            Research Project Agenda         2. Collecting Data         3. Appointing Expert Advisory Panels         4. Supporting Patient and Consumer Representatives         5. Establishing a Methodology Committee         6. Providing a Peer Review Process for Primary Research         7. Releasing Research Findings         8. Adopting Policy         9. Submitting Annual Reports      D. Institutional Design, Governance, and Administration         1. Administration         2. Board of Governors         3. Financial and Governmental Oversight         4. Financial Credibility and Access      E. Dissemination and Building Capacity for Research         1. General Functions         2. Other Requirements and Obligations         3. Researcher Training         4. Data for Research      F. Limitations on the Use of Comparative Effectiveness Research      G. Establishment and Funding of the Patient-Centered Outcomes         Research Trust Fund         1. Establishment of the Patient-Centered Outcomes Research            Trust Fund         2. Uses of Trust Funds         3. Financing the Patient-Centered Outcomes Research            Trust Fund IV. CAN THE COMPARATIVE EFFECTIVENESS INITIATIVE SUCCEED?     A. Political Fallout from Federal Standard Setting     B. The Conundrum of Cost Effectiveness     C. The Private Sector Does the Heavy Lifting     D. The Ideal Structure and Governance of Federal Efforts V. CONCLUSIONS 

The Patient Protection and Affordable Care Act (PPACA), as amended by the Health Care and Education Reconciliation Act of 2010, initiated comprehensive health reform for the healthcare sector of the United States. PPACA includes strategies to make the American healthcare sector more efficient and effective. PPA CA's comparative effectiveness research initiative and the establishment of the Patient-Centered Outcomes Research Institute are major strategies in this regard. PPACA's comparative effectiveness research initiative is one in a long line of federal initiatives to address the rising costs of healthcare as well as to obtain better value for healthcare expenditures. The key question is whether the governance and design features of the institute that will oversee the initiative will enable it to succeed where other federal efforts have faltered. This Article analyzes the federal government's quest to ensure value for money expended in publically funded healthcare programs and the health sector generally. This Article will also analyze what factors contribute to the possible success or failure of the comparative effectiveness research initiative. Success can be defined as the use of the findings of comparative effectiveness to make medical practice less costly, more efficient and effective, and ultimately, to bend the cost curve.

I. INTRODUCTION

The Patient Protection and Affordable Care Act (PPACA), (1) as amended by the Health Care and Education Reconciliation Act of 2010, (2) initiated comprehensive health reform for the healthcare sector of the United States. In addition to increasing access to healthcare coverage through the expansion of public programs and reform of the private health insurance market, PPACA has several initiatives to improve the quality and control the cost of healthcare services.

To make the PPACA coverage expansions affordable, PPACA has some strategies to make the American healthcare sector more efficient and effective. PPACA's comparative effectiveness research initiative is a major strategy in this regard. Congress intended for the comparative effectiveness research initiative to develop hard information about the comparative effectiveness of different medical products and services. This initiative is one of a long line of federal initiatives to address the rising costs of healthcare, as well as obtain better value for healthcare expenditures. The key question is whether the governance and design features of the institute that PPACA created to oversee the initiative will enable it to succeed where other federal efforts have faltered.

This Article analyzes the federal government's quest to ensure value for money expended in publically funded healthcare programs and the health sector generally. This Article will first review the origins of each initiative, the course of its implementation, and the reasons for its ultimate success or failure. Then, this Article will analyze which factors contribute to the possible success or failure of the comparative effectiveness research initiative. This Article asserts that the initiative is successful only if the findings of comparative effectiveness research are used to make medical practice less costly, more efficient and effective, and ultimately, to "bend the curve" for health sector costs.

II. THE ROAD TO COMPARATIVE EFFECTIVENESS RESEARCH

The federal leadership in comparative effectiveness research is possible because of the infrastructure to support and assess research in the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Following World War II, the federal government greatly expanded its commitment to biomedical research. (3) An important result of this funding was the creation of academic medical centers that collectively comprise perhaps the greatest research enterprise in the world. (4) Congress established modern safety and efficacy regulations for pharmaceutical products in the Drug Amendments of 1962, (5) and then regulated medical devices and technology with the Medical Device Amendments of 1976. (6)

In 1965, Congress enacted and the President signed the Social Security Amendments of 1965 that established the Medicare and Medicaid programs. (7) These programs provided publicly funded health insurance coverage for the elderly, the disabled, and some poor. The inauguration of the Medicare and Medicaid programs transformed the federal role in healthcare by making the federal government responsible for paying for the healthcare of a significant portion of the U.S. population.

A. EARLY EFFORTS AT TECHNOLOGY ASSESSMENT

Shortly after implementation, the cost of the Medicare program greatly exceeded prior estimates. (8) Similarly, Medicaid expenditures rose at unexpected levels, putting great pressure on state budgets. (9) Early on, social science researchers recognized the inflationary role of health insurance in healthcare cost inflation. (10)

One cause of the cost inflation was the incredible medical advances following World War II. (11) The development of pharmaceuticals and medical devices for the diagnosis and treatment of illness and injury was been monumental. (12) Yet it soon became apparent that medical advances and new medical technology were major factors in the rising cost of medical care. (13)

By the 1970s, many policy makers, scientists, and other observers were concerned about the federal government's ability to make sound decisions about new technology. (14) In 1972, Congress enacted the Technology Assessment Act of 1972 and established the Congressional Office of Technology Assessment (OTA) to provide "unbiased information concerning the physical, biological, economic, social, and political effects" of technological applications. (15) Over the years, OTA conducted numerous studies on healthcare technologies. (16) In the mid-1990s, the Republican-controlled

Congress declined to appropriate funding for OTA, and the OTA closed in 1996.

In the 1970s, on the theory that new medical technologies were a contributing factor to the escalating costs of medical care, the federal government undertook efforts to assess the effectiveness of new medical technologies. (17) In 1978, Congress established the National Center for Health Care Technology within the Public Health Service. (18) The Center conducted and sponsored assessments of healthcare technologies and coordinated such efforts within the U.S. Department of Health, Education and Welfare (now the U.S. Department of Health and Human Services (DHHS)). (19) The Center also evaluated new technology for purposes of determining whether Medicare should cover the new technology. (20) Opposed by the medical device industry and the American Medical Association, (21) the Reagan Administration eliminated the Center's budget in 1982, and the Center closed. DHHS continued its technology assessment activities in the Public Health Service through the Office of Health Technology Assessment (OHTA). (22) Despite these setbacks for federal efforts at technology assessment, interest in how to effectively assess medical technology remains. (23)

B. THE ASCENDENCY OF HEALTH SERVICES RESEARCH

Health services research developed largely to explain and tame the cost of inflation in healthcare services as well as to improve the quality of and access to these services. In 1969, Congress established the National Center for Health Services Research and Development (NCHSR) in the Department of Health, Education, and Welfare. (24) Academy Health, the primary professional association for health services researchers, defines health services research as follows:

   Health services research is the multidisciplinary field of    scientific investigation that studies how social factors, financing    systems, organizational structures and processes, health    technologies, and personal behaviors affect access to health care,    the quality and cost of health care, and ultimately our health and    well-being. Its research domains are individuals, families,    organizations, institutions, communities, and populations. (25) 

In the 1980s, spurred on by health services research that indicated that little was known about whether expensive medical procedures were more efficacious than less expensive treatment approaches, medical researchers and third-party payers promoted outcome measures as the appropriate indicators of quality in quality assurance and improvement activities. (26) Health services researchers demonstrated that not all costly medical procedures are more effective than less costly ones. (27)

In addition, the Health Care Financing Administration (HCFA), the predecessor agency of the Centers for Medicare and Medicaid Services (CMS) that administers said programs, had a strong research function that conducted research and demonstrations. (28) In the mid-1980s, HCFA launched an aggressive program of research on outcomes of care that would serve as the basis of medical practice guidelines and even coverage policy for federal health insurance programs. (29)

Extensive health services research has also demonstrated that physicians and other providers tended to provide more healthcare than often necessary. (30) The work of Dr. John Wennberg and colleagues has demonstrated that variation in the practice of medicine is great with unexplained variation in services among different geographic areas for the same conditions. (31) Dr. Wennberg and colleagues established the Dartmouth Atlas Project, which uses Medicare data to analyze utilization of healthcare services in national, regional, and local markets, as well as individual hospitals and affiliated physicians. (32) This body of research is an important inspiration for the recent comparative effectiveness research initiatives.

In the Omnibus Budget Reconciliation Act of 1989 ("OBRA '89"), Congress established the Agency for Health Care Policy and Research (AHCPR). (33) The purpose of the Agency was "to enhance the quality, appropriateness, and effectiveness of health care services, and access to such services, through the establishment of a broad base of scientific research and through the promotion of improvements in clinical practice and in the organization, financing, and delivery of health care services." (34)

The statute charges the Agency with conducting and supporting research, demonstration projects, evaluations, training, guideline development, and dissemination of information, on healthcare services and systems for the delivery of such services. (35) Figure 1 presents the subject matter of these activities. The Agency also had to give special attention to the delivery of healthcare services in rural areas and to low-income groups, minority groups, and the elderly. (36) The Agency could also fund independent research centers in not-for-profit entities to conduct these activities. (37) AHCPR also had important dissemination responsibilities. (38)

Figure 1  Subject Matter of AHCPR Research and Other Activities  * The effectiveness, efficiency, and quality of healthcare services;  * The outcomes of healthcare services and procedures;  * Clinical practice, including primary care and practice-oriented research;  * Health technologies, facilities, and equipment;  * Healthcare costs, productivity, and market forces;  * Health promotion and disease prevention;  * Health statistics;  * Healthcare and epidemiology; and  * Medical liability.  OBRA '89 [section] 6103(a) (codified as amended Public Health Service Act [section] 902(a), 42 U.S.C. [section] 299a(a)(1-8)). 

AHCPR replaced the NCHSR and also assumed the technology assessment function of the Public Health Service. (39) AHCPR had to promote the development and application of technology assessments through the activities listed in Figure 2. (40) In specific technology assessments, AHCPR needed to consider "the safety, efficacy, and effectiveness, and, as appropriate, the cost-effectiveness, legal, social, and ethical implications, and appropriate uses of such technologies, including consideration of geographic factors." (41)

Figure 2  Activities to Promote the Development and Application of Technology Assessments  * Identifying needs in, and establishing priorities for, the assessment of specific healthcare technologies;  * Developing and evaluating criteria and methodologies for healthcare technology assessment;  * Conducting and supporting research on the development and diffusion of healthcare technology;  * Conducting and supporting research on assessment methodologies; and  * Promoting education, training, and technical assistance in the use of healthcare technology assessment methodologies and results.  OBRA '89 [section] 6103(a) (codified as amended Public Health Service Act [section] 904(a)(1-5), 42 U.S.C. [section] 299a-2(a)(1)-(5)). 

AHCPR also had to make recommendations on whether the Medicare program should pay for specific medical technologies and/or any conditions and requirements attending reimbursement for the technology. (42) In making these recommendations, AHCPR had to consider the "safety, efficacy, and effectiveness, and, as appropriate, the cost-effectiveness and appropriate uses of such technologies." (43) It is noteworthy that "cost-effectiveness" was a criterion for AHCPR technology assessments, as this criterion has been so controversial in making Medicare coverage policy and in the current comparative effectiveness research initiative established under PPACA. (44)

OBRA '89 established several other notable programs. Perhaps the most well known program is the Forum for Quality and Effectiveness in Health Care. (45) Pursuant to procedures and requirements specified in the statute, (46) the Program had to arrange for the development and periodic review of the following:

(1) clinically relevant guidelines that may be used by physicians, educators, and health care practitioners to assist in determining how diseases, disorders, and other health conditions can most effectively and appropriately be prevented, diagnosed, treated, and managed clinically; and

(2) standards of quality, performance measures, and medical review criteria through which health care providers and other appropriate entities may assess or review the provision of health care and assure the quality of such care. (47)

The Forum was designed to facilitate guideline development through the convocation of expert panels and the formal recognition of guidelines developed by other appropriate groups. (48)

The second important program in health services research was the research program on Outcomes of Health Services and Procedures under Title XI of the Social Security Act. (49) This program conducted and supported research on "the outcomes, effectiveness, and appropriateness of health care services and procedures in order to identify the manner in which diseases, disorders, and other health conditions can most effectively and appropriately be prevented, diagnosed, treated, and managed clinically." (50) Research had to ensure that the procedures for developing treatment-specific or condition-specific practice guidelines reflected the needs and priorities of the Medicare and Medicaid programs. (51) The program also had to conduct and support "evaluations of the comparative effects, on health and functional capacity, of alternative services and procedures utilized in preventing, diagnosing, treating, and clinically managing diseases, disorders, and other health conditions." (52)

Under this authority, HCFA established the interdisciplinary Patient Outcome Research Teams (PORTs), which evaluated specific procedures widely used by Medicare beneficiaries. (53) The research program was also responsible for establishing initial medical practice guidelines for high-volume Medicare services. (54) HCFA had to use these guidelines in the Medicare program to "improve the quality, effectiveness, and appropriateness of care" for Medicare beneficiaries. (55) Its statutory mandate clearly envisioned the development of guidelines that Medicare programs and other third-party payers would use to determine coverage of benefits and payment levels.

AHCPR had a very turbulent history, especially with the advent of Republican majorities in Congress in 1994. (56) Following the release of a controversial guideline for the treatment of low back pain, (57) several members of Congress sought to terminate funding for AHCPR. (58) Further, a manufacturer of devices used to treat low back pain sued the Agency for alleged violations of the Federal Advisory Committee Act (59) in connection with the Low Back Pain Panel that developed the guideline. (60) Congress ultimately spared funding for the Agency on the condition that the Agency only focus on quality measurement and improvement in the future. (61)

In the Health Care Research and Quality Act of 1999, Congress established the Agency for Healthcare Research and Quality (AHRQ) to replace the controversial AHCPR. (62) The mission of the new Agency was "to enhance the quality, appropriateness, and effectiveness of health services, and access to such services, through the establishment of a broad base of scientific research and through the promotion of improvements in clinical and health system practices, including the prevention of diseases and other health conditions." (63)

Figure 3 presents specific research issues that the Agency was to address. AHRQ's mission was also to support the synthesis and dissemination of available scientific evidence for use by patients, consumers, practitioners, providers, purchasers, policy makers, and educators, (64) as well as to support initiatives to advance private and public efforts to improve healthcare quality. (65) The statute imposed requirements for research with respect to rural and inner-city areas and priority populations. (66)

In executing its mission, the director of AHRQ has specific statutory duties. These duties include: (1) conducting and supporting research, evaluations, and training; (2) supporting demonstration projects, research networks, and multidisciplinary centers; (3) providing technical assistance; and (4) disseminating information on healthcare and systems for the delivery of such care. (67) Agency research and other activities must address the issues listed in Figure 3. The statute requires that the Agency's activities be "appropriately coordinated" with experiments, demonstration projects, and other related activities conducted with respect to the Medicare, Medicaid, and State Children's Health Insurance (SCHIP) programs. (68)

Figure 3  Subject Matter of AHRQ Research and Activities Research Issues Addressed as Part of the Mission of AHRQ*  * The development and assessment of methods for enhancing patient participation in their own care and for facilitating shared patient-physician decision making;  * The outcomes, effectiveness, and cost-effectiveness of healthcare practices, including preventive measures and long-term care;  * Existing and innovative technologies;  * The costs and utilization of, and access to, healthcare;  * The ways in which healthcare services are organized, delivered, and financed and the interaction and impact of these factors on the quality of patient care;  * Methods for measuring quality and strategies for improving quality; and  * Ways in which patients, consumers, purchasers, and practitioners acquire new information about best practices and health benefits, and the determinants and impact of their use of this information.  Required Subject Matter of AHRQ Activities**  * The quality, effectiveness, efficiency, appropriateness, and value of healthcare services;  * Quality measurement and improvement;  * Outcomes, cost, cost-effectiveness, and use of healthcare services and access to such services;  * Clinical practice, including primary care and practice-oriented research;  * Healthcare technologies, facilities, and equipment;  * Healthcare costs, productivity, organization, and market forces;  * Health promotion and disease prevention, including clinical preventive services;  * Health statistics, surveys, database development, and epidemiology; and  * Medical liability.  * Health Care Research and Quality Act of 1999 [section] 2(a) (codified as amended Public Health Service Act [section] 902(a)(1-8), 42 U. … 

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