BCBSA Statement on Comparative Effectiveness Hearing, June 12, 2007

June 12, 2007

Statement for the Record to: Committee On Ways And Means

Subcommittee On Health

U.S. House Of Representatives

On Comparative Clinical Effectiveness Information

Submitted by: Blue Cross and Blue Shield Association

Introduction

The Blue Cross and Blue Shield Association (BCBSA) appreciates the opportunity to share with the Ways and Means Subcommittee on Health our recommendation that Congress charter an independent institute (Institute) to support comparative clinical effectiveness research. The proposed Institute is key to tackling the root causes of our nation's healthcare affordability and coverage problems – by empowering providers and consumers with the knowledge they need to make appropriate evidence-based healthcare decisions.

The Blue Cross and Blue Shield Association is made up of 39 independent, community-based and locally operated Blue Cross and Blue Shield Plans that collectively provide healthcare coverage to more than 98 million – nearly one-in-three – Americans. For more than 75 years, BCBS Plans have been committed to improving the quality, safety and affordability of healthcare for all Americans.

BCBSA commends Chairman Stark and Ranking Member Camp for focusing attention on the need for greater medical evidence to guide healthcare decisions. We strongly believe there must be collaborative public and private support for research to determine what medical treatments best deliver positive outcomes for patients. BCBSA looks forward to working with the Subcommittee and all healthcare stakeholders to support evidence-based medicine that will help Americans lead healthier lives.

The following is BCBSA’s proposal to Congress to establish an independent, private Institute based on four key principles:

1. Stable funding for the Institute should be ensured by asking all healthcare payers to contribute.
2. The Institute should support a broad range of research, especially clinical trials.
3. Significant education and incentives are needed to ensure new research improves clinical practice.
4. The new Institute should be chartered as an independent, private entity.

Background

Much of healthcare delivered today lacks a basis in evidence. Researchers and policymakers agree there often is not enough information on what medical treatments work best. Every day, providers and patients must make important decisions without knowing how different treatments compare. The end result: Patients pay more for sub-optimal care. One study estimates up to 30 percent of healthcare spending pays for ineffective, inappropriate, or redundant care.ⁱ Another study estimates that only 54 percent of acute care and 56 percent of chronic care provided by physicians conforms to medical literature.ⁱⁱ

The absence of good evidence can result in harmful care. For example, in the mid to late 1980s, some studies provided limited evidence that autologous bone marrow transplant/high-dose chemotherapy (ABMT/HDC) was more effective than conventional chemotherapy for treating metastatic breast cancer. However, after rigorous clinical trials were performed in the 1990s, it became apparent that conventional chemotherapy was superior to ABMT/HDC. Thus, some 30,000 women were unnecessarily subjected to ABMT/HDC. ABMT/HDC is estimated to have caused around 600 premature deaths.

The ABMT/HDC case – with the approximately 10-year delay in producing credible comparative information – shows the negative impacts that can occur due to insufficient research conducted in critical areas. In particular, expense is a major factor why not enough comparative clinical trials are being done – for example, the recent COURAGE study (comparing coronary stents versus drugs) cost approximately $33.5 million, and the ALLHAT study (comparing different antihypertensive and lipid-lowering drugs) cost more than $83 million. While several federal agencies conduct some health services research, none has a strong focus on supporting comparative clinical trials. For example, the National Institutes of Health (NIH) do not have a significant focus on comparative trials, and the Agency for Healthcare Research and Quality’s (AHRQ) modest budget is generally spent on literature reviews and other studies. Furthermore, many of the clinical trials supported by the private sector focus on establishing product efficacy for purposes of Food and Drug Administration (FDA) approval and typically do not involve comparisons to other treatment options.

Experience teaches us that the medical literature often lacks comparative data on multiple possible treatments for similar diseases or conditions. The absence of such data, as well as the frequent use of available therapies for “off-label” indications, can lead such treatments being considered investigational or experimental.

BCBSA has been a leader in providing evidence-based technology assessments since 1985, when BCBS Plans founded and began to fund the Technology Evaluation Center (TEC). In addition to serving BCBS Plans, TEC has provided services to clients such as the Centers for Medicare and Medicaid Services (CMS) and Kaiser Permanente, as well. TEC has been one of AHRQ's Evidence-based Practice Centers (EPCs) since 1997. (AHRQ's EPCs are charged with drafting reports on specified healthcare conditions that synthesize existing research, promote evidence-based medicine, and identify research gaps.)

Through the years, TEC has produced over 400 assessments that evaluate medical procedures, drugs, biological products, and devices – about 15-20 assessments each year. Nine of these reports have been produced for AHRQ. These assessments take into account existing clinical and scientific evidence and evaluate whether a technology improves health outcomes such as length of life, quality of life, and ability to function. A three-year rolling inventory of TEC assessments is available for providers and consumers to download on the internet at www.bcbs.com/tec.

TEC assessments are researched and drafted by a team of experienced physicians and doctorate-level scientists who are supported by professionals in pharmacology, nursing, statistical methods, and library science. Ultimately, a 19-member Medical Advisory Panel has final scientific and clinical authority and accountability for all TEC assessments. The Medical Advisory Panel is comprised of independent, nationally recognized experts in technology assessment, clinical research, and medical specialties, and includes members with faculty appointments at universities such as Harvard, Stanford, and Johns Hopkins.

BCBSA is very proud of TEC's contribution to evidence-based medicine, but our Plans and their providers agree that there is a need for much greater investment in comparative effectiveness research, especially expensive clinical research. BCBSA believes federal legislation chartering a new independent Institute to support comparative effectiveness research is essential to improving both healthcare quality and the affordability of health coverage. BCBSA supports the following four principles for the development of an independent comparative effectiveness Institute.

Principle 1: Ensure Stable Funding By Asking All Payers To Contribute

The most significant challenge in creating a new Institute is ensuring a stable and significant funding source. BCBSA believes the Institute should be funded by assessments on all private coverage (insured and self-funded) and public healthcare coverage such as Medicare, Medicaid, and other government programs. Broad-based funding ensures that all payers contribute to the development of new information that benefits the healthcare system as a whole.

Furthermore, BCBSA recommends an initial budget of approximately $500 million annually. In the future, as the Institute's work impacts clinical practice and improves healthcare quality, and there are assurances that adequate infrastructure is in place to support additional research work, consideration should be given to whether budget increases are warranted.

BCBSA's commitment to all-payer funding is contingent on an Institute that meets key principles – for instance, one that is an independent entity, with appropriate private stakeholders on the Board, and where an appropriately set budget is fairly funded from government programs (e.g., Medicare and Medicaid) as well as private payers.

Principle 2: Support A Broad Range Of Research, Especially Clinical Trials

The Institute should prioritize and fund a variety of research – including clinical trials, cohort studies, literature reviews, and other studies – evaluating the comparative clinical and cost effectiveness of different procedures, drugs, devices, and biologics. A broad research role is important because:

• No government agency (e.g., NIH, AHRQ, etc.) currently has a significantly funded and dedicated focus on comparative clinical research; and
• The great costs of comparative clinical trials make it unlikely that other entities will have the resources to sponsor them.

The Institute also should encourage improved efficiency by collaborating with the institutions that receive comparative research contracts to identify best practices and efficiencies to maximize research dollars.

Principle 3: Ensure Research Improves Clinical Practice Through Education, Incentives

The Institute should ensure that new comparative information is disseminated to providers, patients, and others. Because experts estimate that medical research currently takes 17 years to be incorporated into practice, it is especially important that this information be provided in formats providers easily can use. The Institute should work with medical societies to develop consensus practice guidelines drawing on the Institute's research, and electronic health records should be required to incorporate these guidelines into their clinical decision support systems.

In addition, the guidelines' evidence-based standards should create rebuttable presumptions concerning the standards of care that providers are expected to meet in medical malpractice cases. Providers that reasonably rely on the guidelines should be given safe harbors for non-economic (e.g., pain and suffering) and punitive damages, even if their use of the guidelines is later judged to be mistaken.

BCBSA believes that the Institute's role should be limited to being a credible source of evidence-based medicine information and that it should not be involved in setting pay-for-performance, coverage, reimbursement, and other policies based on its research. These kinds of decisions should be made by public and private payers themselves. Accordingly, Medicare and other public programs should consider the relevance of the Institute's research as part of their policy development processes.

Principle 4: Ensure An Independent Entity

The Institute should be structured as an independent, not-for-profit entity. The Institute should be governed by a board of experts who bring the perspectives of a wide range of stakeholders (e.g., providers, consumers, government, health insurers, employers, and, manufacturers). This structure is intended to achieve two related goals:

• The Institute should be accountable to the public, given its mission and large budget; and
• The Institute should be protected from political pressures so it can establish and maintain a reputation as a credible source for information on evidence-based medicine.

The Institute may contract with existing entities (e.g., AHRQ) to facilitate research.

Conclusion

The proposed Institute represents a major step in confronting our nation’s healthcare challenges at their roots. Public and private support for greater comparative clinical research can help ensure our healthcare resources go toward the most effective care.

BCBSA thanks the Chairman and the Ranking Member for holding a hearing on this important issue.

i Fisher, E., Wennberg, D., et al., The Implications of Regional Variations in Medicare Spending: Part 2, Health Outcomes and Satisfaction with Care, Annals of Internal Medicine 2003; 138:288-98.

ii McGlynn E.A., et al., The Quality of Care Delivered to Adults in the United States, New England Journal of Medicine 2003; 348:2635-45.