Comparative Effectiveness: Can We Get Better Health Value for What We Spend?

1. Comparative Effectiveness: Can We Get Better Health Value for What We Spend? Wilhelmine Miller, MS, PhD Department of Health Policy GW School of Public Health and Health Services September 23, 2008 National Congress on Health Reform Workshop Session 1A

2. Overview • Comparative effectiveness research in the context of health reform: goals and rationale • The status quo • Elements of a national program of comparative effectiveness (CE) research (IOM recommendations) • Pending CE legislation • Challenges and opportunities for integrating CE information in a reformed health care enterprise

3. Reforming the health care enterprise: 3 aspects, intertwined • Universal access • 45.7 million Americans without coverage • Quality improvement • Americans receive recommended services just over half of the time • Cost containment • Medicare and Medicaid per capita spending is growing 2.5 percentage points faster than per capita GDP; US per capita health spending is 2.4 times that of all other developed countries

4. Why are extensions of coverage essential for reform along the dimensions of quality and cost? • Uninsured and un-served people are not captured within payer-based systems of accountability that promote quality care • Efforts to reform payment policies systematically are undermined by the legitimate need for implicit cross-subsidies and financial devices to pay for uninsured health care

5. What are some goals of comparative effectiveness research? • Rigorously evaluate the impact of alternative diagnostic and treatment options • Identify the best treatments for particular patients with a given condition • Assess the health benefits of alternative health interventions in relation to their costs

6. What can comparative effectiveness research contribute? • Improved quality of care through more appropriate and effective services • Better health outcomes for a given level of spending on health care • Reduced variation in health services and spending across geographic areas • Better-informed patients and consumers

7. What should a national CE research effort look like? • What is involved in CE research and dissemination? • What do we in the US do now? • How much (more) CE research do we need? • Who in the world does this well?

8. Body of evidence Systematicreviews • Policy applications • Practice guidelines • Performance measures • Insurance coverage • Consumer information RCTs Cohort studies Case control studies Registries Administrative data Evidence-Based Practice Centers Proprietary firms • USPSTF • Prof’l groups • NIH • VA • CMS • BC/BS • Consumers

9. Strengths of US CE Activities and Capacity • Well-developed systematic review methods • A network of professionals skilled in systematic reviews • Pluralistic, close to the ground • Some excellent models for transparent, rigorous guideline development • Influential users of guidelines (e.g., coverage decisions, performance measures)

10. Problems with the Status Quo • Extensive duplication of efforts by insurers and private groups is inefficient • Potential conflicts of interest in assessing evidence and promulgating guidelines • Systematic reviews and guidelines often lack scientific rigor • Confusing advice: National Guidelines Clearinghouse contains more than 2000 guidelines by 350 organizations • Difficult for users to see the connection between the evidence and clinical recommendations

13. Duplication of Efforts Of 20 services* - 14 evaluated by all 7 groups - 17 evaluated by 5 of 7 groups - 5 evaluated by AHRQ * 2006 activities

14. Current Investments in CE Research • AHRQ’s Effective Healthcare Program • Authorized by Section 1013 MMA (2003) • $30 million annual budget • NIH, FDA-required pre-approval studies, VA, Medicare “coverage with evidence development (CED)” • Health plans, insurers • Professional and disease-specific organizations

15. The National Conversation on Comparative Effectiveness Research • We need more head-to-head RCTs to establish comparative clinical effectiveness • A national program to develop and disseminate information on CE should be established (IOM, MedPAC, AHIP) • Agency must be insulated from political pressures • CE research should be publicly funded or jointly funded by public and private insurers • The program could cost as little as $50 million/year if it did not fund new clinical trials—OR—if it did fund new research, could spend $300-$800 million/year (AHIP Rx: 1/10 of 1 percent of national health care spending)

16. We Have Good International Models • U.K.: National Institute for Health and Clinical Excellence (NICE) • ~$60 million annual budget • Clinical and cost effectiveness of new and existing interventions • Australia: Pharmaceutical benefits and medical services advisory committees • Clinical and cost effectiveness of new drugs, technologies and procedures • Canada: Canadian Agency for Drugs and Technology in Health (CADTH) • coordinates clinical and economic assessments and makes coverage recommendations to Provincial plans

17. Process and organization of a national comparative effectiveness research program • The IOM report, Knowing What Works in Health Care: A Roadmap for the Nation, recommends • Approach to identifying highly effective clinical services • Process to evaluate evidence about clinical effectiveness • Organizational framework for using evidence reports to make recommendations • The consensus report does not • Consider the use of cost-effectiveness analysis • Recommend an organizational locus for the new program • Recommend funding levels for clinical effectiveness research

18. IOM Committee Recommendations • A single national entity should have the authority, overarching responsibility, resources, and adequate capacity to ensure that credible, unbiased information about clinical effectiveness is produced. • An appointed Clinical Effectiveness Advisory Board should oversee the program and be constituted to • minimize bias due to conflict of interest • represent diverse public and private sector expertise and interests • Program should develop standards to minimize bias due to conflicts of interest for • priority setting • evidence assessment • guidelines development

19. Building a Foundation for Knowing What Works Program activities: • Set priorities for, fund, and manage systematic reviews of clinical effectiveness and related topics • Develop a common language and standards for conducting systematic reviews of evidence and generating clinical guidelines and recommendations • Provide a forum for addressing conflicting guidelines and recommendations

20. Setting Priorities • A standing advisory committee should identify high-priority topics for systematic reviews of clinical effectiveness • Priority setting process should be open, transparent, efficient, and timely • Priorities should reflect • the potential to improve health across the life span • reduce the burden of disease and health disparities • eliminate undesirable variation • consider economic factors, such as costs of treatment and economic burden of disease • Advisory committee membership should include a broad mix of expertise and interests and be chosen to minimize bias due to conflicts of interest

21. Assessing Evidence • Develop evidence-based methods standards for systematic reviews, including a common language for characterizing the strength of evidence • Fund reviewers only if they commit to and consistently meet these standards • Invest in advancing the scientific methods underlying the conduct of systematic reviews and update standards for funded reviews as appropriate • Assess the capacity of the research workforce to meet the needs for systematic reviews • Expand training opportunities in systematic review and comparative effectiveness research methods as appropriate

22. Developing Clinical Practice Guidelines • Groups developing clinical guidelines or recommendations should • adhere to evidence and bias/conflict of interest standards • document their adherence • make documentation publicly available • Panels should include a balance of competing interests and diverse stakeholders, publish conflict of interest disclosures, and prohibit voting by members with material conflicts to minimize bias

23. Using Clinical Practice Guidelines • Who should preferentially use guidelines developed according to standards of evidence and without material conflicts of interest? • Clinical training programs • Clinicians and providers • Public and private insurers • Purchasers • Accrediting organizations • Performance measurement groups • Patients and consumers

24. Pending CE legislation • Comparative Effectiveness Research Act of 2008 • Introduced by Senators Baucus and Conrad July 31 • Establishes independent private, nonprofit corporation (“Health Care CE Research Institute”) • Governed by 21-member Board appointed by Comptroller General and performance reviewed by GAO • Research may be conducted under contract with public and private agencies and is subject to peer review • Requires public comment periods and public forums • Funding from general revenues, Medicare Trust Fund, insurers and self-insured plans • After 5-year ramp up, annual funding of $300 million

25. Challenges for integrating CE information into a reformed (or any!) US health care enterprise • Changing clinicians’ behavior, even with better and more accessible CE information • Continuing pressure from commercial sponsors, patients, and clinicians to do more rather than less and to adopt new technologies • Reconciling stakeholder representation in CE activities with unbiased and un-conflicted assessments & decisions • Conducting CE research and making recommendations in a timely fashion

26. Opportunities that CE research offers in health care reform • Potential cost savings • CBO estimated system-wide savings of ~$6 billion over 10 years for last year’s HR 3162 • Commonwealth Fund analysis estimates more than 50X that amount in 10-year savings ($368 billion) • Good models and prototype programs (e.g., AHRQ’s Effective Health Care program) • Economies in production of CE information could be immediate with reduced duplication of new technology analyses • Cost data should be collected along with clinical outcomes (even if CEA is precluded in the first round) • CE information is a “two-fer”, with potentially positive effects on 2 of the 3 legs of health reform, quality and cost