Presentation on Comparative Effectiveness Research at University of Tennessee, Knoxville, September 2, 2010

1. Comparative Effectiveness Research and Health Care Reform Graduate Seminar in Public Health University of Tennessee, Knoxville Rick Mathis, PhD rsmathis@me.com

2. Define Comparative Effectiveness Research The Policy Context of CER Current Efforts Thoughts about the Future Focus

3. Comparative effectiveness research (CER) is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers and policy makers to make informed decisions that will improve health care at both the individual and population levels. National Priorities for Comparative Effectiveness Research, Institute of Medicine Report Brief 2009 Definition CER

4. “None of us agree on what comparative effectiveness research actually is, but we all agree that it will cost about $5 billion to do it.” Jack Rowe, former CEO of Aetna Definition of CER - 2008

5. Clinicians – what should Iprescribe Patients and caregivers – what care should I seek Payers – what should Ireimburse Policy makers – what research should Iencourage; what best improves public health Who is Concerned about CER

6. Prostate cancer – what is the best treatment; is robotic surgery the best approach; how and when should radiation be used; how should test results be interpreted Wound care – how to improve the evidence and assess new treatments Cardiac imaging – when should imaging be ordered and for whom Schizophrenia – recent drugs are more expensive but may not represent improvements Hypertension – earlier treatments are cheaper and as effective Treatment issues

7. The Evidence Paradox How to reconcile: 18,000+ RCTs are published each year Tens of thousands of non-experimental studies Many systematic reviews, health technology assessments, clinical guidelines conclude that the “available evidence is limited or studies are poor quality”

8. Research subjects are highly selected Research settings are not typical of settings in which care is usually delivered Missing or incorrect comparators Physiologic or surrogate outcomes, not functional status, long term impacts Statistical problems: sample size, bias, etc. Common Gaps in Evidence

9. Will CER lead to rationing? Will treatments beneficial to minorities be excluded? Will treatments for rare conditions be excluded? What about personalized medicine? Will innovation be stifled? What about cost effectiveness? Supported by both parties but there are concerns

10. Legislate against the use of CER in benefit design Include diverse groups at all phases of studies Look at treatment effects on subgroups of patients Make it “patient centered” The Response

11. Decision makers (patients, consumers, clinicians, payers, policy makers) should have greater influence in guiding the activities of the clinical research enterprise 11 The CER Hypothesis

12. The American Recovery and Reinvestment Act of 2009 allocated $1.1 billion for comparative effectiveness research AHRQ: $300 million NIH: $400 million Secretary Health and Human Services: $400 million Institute of Medicine identified the top priority conditions and treatment modalities to be studied (June 30, 2009) Federal Coordinating Council to coordinate comparative effectiveness research across agencies First 6 months after ARRA

13. Atrial Fibrillation treatments Hearing loss in children Primary prevention vs. clinical treatment in preventing falls in older adults Effectiveness of medical home model in treating chronic conditions Inflammatory disease Prostate cancer Some of IOMs Top Quartile Recommendations

14. Patient-Centered Outcomes Research ‘The terms ‘comparative clinical effectiveness research’ and ‘research’ mean research evaluating and comparing health outcomes and the clinical effectiveness, risks, and benefits of 2 or more medical treatments and services There is authorized to be established a nonprofit corporation, to be known as the Patient-Centered Outcomes Research Institute which is neither an agency nor establishment of the United States Government” Patient Protection and Affordability Act of 2010

15. Recently Passed Reform Legislation:From CER to PCORI Establishes a Patient-Centered Outcomes Research Institute to “assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis that considers variations in patient subpopulations, and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of the medical treatments, services, ... "

17. Establishes research project agenda, carries out research agenda, contracts for the management of funding, appoints ad hoc expert advisory panels, etc.

18. Board of Directors includes Director of AHRQ, NIH, and 17 members representing patients, consumers, physicians, and private payers

19. Establishes research priorities and a project agenda based on prevalence and burden of diseases

21. Methodological standards for research. Such methodological standards shall provide specific criteria for internal validity, generalizability, feasibility, and timeliness of research and for health outcomes measures, risk adjustment, and other relevant aspects of research and assessment with respect to the design of research Include input from all relevant experts, stakeholders, and decision makers, and shall provide opportunities for public comment 18 Research Methods in legislation

22. Example of CER Study Currently Underway

23. Center forComparative Effectiveness Research in Cancer Genomics (CANCERGEN) Fred Hutchinson Cancer Research Center Southwest Oncology Group University of Washington Center for Medical Technology Policy

24. “…insufficient evidence to make a recommendation for or against the use of tumor gene expression profiles…” “The EWG encourages further development and evaluation of these technologies.” Recommended 9 topics for further research EGAPP on Genetic Tests in Breast Cancer (Jan 2009)

26. Integrate comparative effectiveness within SWOG to facilitate rapid design and implementation of CER trials in priority areas.

28. Defining evidence standards where randomized controlled trials may not be practical (pragmatic clinical trials) Registries Patient reported outcomes Stakeholder meetings Coverage with Evidence Development Additional CER Approaches

29. Value Based Benefits Formulary development Pay for performance Healthcare disparities Preauthorization Potential Health Plan Uses

30. AHRQ Effective Care Program http://www.effectivehealthcare.ahrq.gov/ Other Studies/Sources

31. Hard to argue against effectiveness Links to value based care, electronic medical records, accountable care organizations What will we be comfortable with as a nation Daniel Callahan: Taming the Beloved Beast: How Medical Technology Costs are Destroying our Health Care System Questions? The Future . . .