Comparative Effectiveness Research

1. Evelyn P. Whitlock, MD, MPH Director, Evidence-based Medicine, TCHR Associate Director, Oregon Evidence-based Practice Center & Scientific Resource Center for the Effective Health Care Program Comparative Effectiveness Research All Center Meeting June 17, 2009

2. Presentation Goal • To explore the recent national focus on Comparative Effectiveness Research (CER) and its relationship to health care reform • To understand how CER opportunities affect TCHR’s research agenda

3. Comparative Effectiveness Research • Generating better information about the costs, risks, and benefits of different treatment options—through research on the comparative effectiveness of those options—and combining this information with incentives to change practice could help reduce health care spending without adversely affecting overall health. Congressional Budget Office report, December 2007 (Peter Orzag, then Director of CBO, now White House Director of Budget)

4. Don’t we know all this already? • For many drugs, the only evidence we have comes from FDA-approval studies • Short-term studies of measured benefits in highly selected patients • Placebo-controlled

5. Other Important Considerations • Patient-centered medicine: must be robust enough to address the patient subgroups that would benefit from treatment • Cost: studies that consider cost-effectiveness and clinical effectiveness would have a greater impact Congressional Budget Office report, December 2007

6. But, before congress could consider and respond to this report, the global economic crash occurred…

7. Bringing on the federal stimulus $$… and a rapid onset of CER activity

8. Why Federal Funding for Comparative Effectiveness Research? • The federal government has a role and stake in comparative effectiveness research • Comparative studies are not generally required for FDA approval as safe and effective • Medical procedures account for more costs than drugs and devices, but approval isn’t required before entering the marketplace

9. Estimated Contributions of Selected Factorsto Long-Term Growth in Real Health CareSpending per Capita, 1940 to 1990 (from CBO 2007 report) Percent

10. Sources of Growth in Projected Federal Spending on Medicare and Medicaid: CBO Projections

11. Why Federal Funding for Comparative Effectiveness Research (cont.) • The federal government has a role and stake in comparative effectiveness research • Private sector has limited incentives for CER—although it funds most clinical effectiveness research • The federal government pays for about 45% of US health expenditures, including care delivered in its own facilities • For similar reasons, CER and technology appraisal are occurring in developing countries around the world

12. CE Research in the American Recovery and Rehabilitation Act (ARRA) 2009 • ARRA allocated $787 billion dollars for recovery-related spending in 2009-2010 • ARRA included $1.1 billion in funding for CER (0.14% of all ARRA) • NIH: $400 million (NIH Challenge and GO grants) • AHRQ: $300 million (late summer/fall 2009) • DHHS: $400 million (informed by IOM and Fed Coord Council on CER reports in June, 2009)

13. That Controversial 0.14% • “Socialized medicine…rationing healthcare… gov’t takeover… getting between you and your doctor… creating waiting in line… loss of choice… denying you what you need when you need it… one-size-fits-all medicine”

14. That Critical 0.14% • We aren’t spending enough on the studies that matter • We aren’t studying what decision-makers (doctors and patients) need to know—what works (best) for whom • A main goal is to counter disparities in access and delivery of care—we already ration care • Cost effectiveness is a necessary part of decision-making in healthcare (American College of Physicians)

15. Health Reform Now—costs and access are affecting more and more Americans The ranks of the uninsured have grown by 8 million people since 2000, with around 46 million Americans currently uninsured in 2007 (U.S. Census Bureau., August 2008) A recent poll found that nearly half of insured U.S. adults said they are concerned that their benefits would be inadequate if they got sick. Among the uninsured, nearly four in 10 named cost as the main reason they don't have health coverage, while 22% said they do not have coverage because they are unemployed or lost their job. Brodie said, "We definitely are seeing that people are being hit hard by the cost of health care," adding, "It's something that bothers people; it worries them. It is the key element of the public's agenda for any kind of health care reform: They want to see their own health care costs go down" (NPR.org, 4/22/09).

16. How to afford more health care? • Focused comparative effectiveness research might help: • Eliminating extreme variations in medical practice could reduce Medicare costs up to 30% • Incorporating information on the relative clinical value into care coverage could improve quality and save money • Other…ideas are being floated daily and hopefully good ones will end up as part of HC reform

17. But, will CER save money? • No one knows for sure • The impact on healthcare spending is hard to estimate and could take more than a decade to materialize • Few other options exist to preserve quality and increase access through containing costs, yet costs are not clearly included

18. Comparative Effectiveness Research—not just more research but more action • Generating better information about the costs, risks, and benefits of different treatment options—through research on the comparative effectiveness of those options—and combining this information with incentives to change practice could help reduce health care spending without adversely affecting overall health. Congressional Budget Office report, December 2007 (Peter Orzag, then Director of CBO, now White House Director of Budget)

19. So what does this mean for TCHR? We’ve got a head start…

20. CER and TCHR’s Future Research Agenda • CER builds on existing TCHR strengths: • Effectiveness research • Evidence synthesis/systematic review • Health services research/clinical epidemiology • Translation of research to practice • Cost/health economics • Addressing policy/practice relevant issues • Engaging stakeholders/users of research

21. Mechanisms for CE Research—Building on TCHR strengths • Systematic reviews • Surveys, claims, and other administrative data • Electronic medical records • Registries • Prospective evaluations • Randomized controlled trials/pragmatic trials • Modeling studies, including cost effectiveness

22. CER Opportunities: Immediate Impacts on TCHR Research • More proposals—increased quick-turnaround funding • Packaging our expertise differently • Comparative Effectiveness Research Unit • Kaiser Permanente Center for Excellence in Effectiveness and Safety

23. CER Opportunities: Expected Longer-Term Impacts • Areas of Growth • CER methodological research • CER agenda development/process research • Modeling/decision analysis/cost-effectiveness • Systematic reviews • Observational studies using Kaiser-wide integrated data • Personalized medicine • Translation into practice • International collaborations

24. A Cautionary Note… • Comparative effectiveness research has always been highly political—and continues to be • History illustrates the short lives of institutions trying to use evidence to improve US healthcare • DHHS National Center for Health Care Technology (1978-1981) • US Congress Office of Technology Assessment (eliminatedin1995) • AHCPR (1989—with change to AHRQ and restriction infundingin 1996)

25. A Health Reform Opportunity… • The current environment may offer the necessary conditions to allow comparative research to realize its potential and facilitatehealth care reform • Comparative effectiveness has enjoyed bipartisan support, although there were efforts to dismantle it in the stimulus package • Other countries have negotiated some of the pitfalls and have lessons to teach us