Evidence-Based Medicine: Implications for Coverage, Practice, and Research

1. Evidence-Based Medicine: Implications for Coverage, Practice, and Research Nuts and BoltsResearch Methods Symposium UT College of Medicine Chattanooga September 29, 2006 Steven Teutsch, MD, MPH, Executive Director Outcomes Research, Merck

2. Agenda • Evolution of medicine and evidence-based medicine (EBM) • The EBM approach and evidence-based decision making • Translating research into practice and practice into research

3. Evolution of Evidence-based Medicine • Early 20th Century • Flexner Report led to dominance of biomedical model of medical care • Mid-Late 20th Century • Rapid growth in biomedical science and new technologies • Physicians primary decision makers • Lack of coherent synthesis of knowledge enables practice variation (Wennberg) • Emergence of Evidence-based Medicine • Appearance of Practice Guidelines and Performance Metrics • Consensus-based (Professional Societies) • Evidence-based (USPSTF, AHCPR, ACP) • NCQA (e.g., HEDIS), Pay-for-Performance

4. End of 20th Century:Health Care Costs Explode • International response • Australia and Ontario introduce Cost Effectiveness as a criterion in formulary decisions • UK establishes NICE • Employs EBM and cost effectiveness to control access to new treatments • Increases credibility of these processes • Recommends, but does not enforce implementation • US response • Rise of Managed Care and Consolidation of Payers • Treatment Guidelines, Preferred Drug Lists/Formularies

5. Early 21st Century – USApplication of EBM and HECON to coverage decisions by States (Medicaid) and Federal Government (Medicare) • Medicare Modernization Act (MMA) • Comparative effectiveness (e.g., EBM) – Section 1013 (AHRQ) • Center for Medicare and Medicaid Services (CMS) • CMS endorsement of health economics in MMA formulary design • National Coverage Decisions • Coverage with Evidence Development • Drug Effectiveness Review Project – (DERP, 17 State Consortium - Medicaid) • Evidence-based Drug Class Reviews • Agency for Healthcare Research and Quality (AHRQ ) • Centers for Research & Education in Therapeutics (CERTs) • Evidence-based Practice Centers (EPCs) • Effectiveness Health Care Program • Comparative Effectiveness (EPCs) • Network of Research Centers (DEcIDE) • Eisenberg Center for Clinical Decisions and Communications (Oregon)

6. The Evidence-Based Approach

7. Evidence-based . . . . • Evidence-based Medicine (EBM) • Patient care based on evidence from the best available studies • Evidence-based Decision Making • EBM extended to include population-based decision making in the form of guidelines and formulary decisions using formal evidence criteria and deliberative processes • Comparative Effectiveness • Subset of EBM which assesses alternative therapies

8. What is Evidence-Based Medicine? “The process of finding relevant information in the medical literature to address a specific clinical problem; the application of simple rules of science and common sense to determine the validity of the information; the application of the information to the clinical question. In short, patient care based on evidence derived from the best available (“gold standard”) studies.” John Last, A Dictionary of Epidemiology, Oxford, 1995

9. Systematic Evidence Reviews • Address a specific problem • Examine highest quality studies • Apply tools critical appraisal to assess studies • Summarize implications of the evidence

10. Contrast with Expert Reviews • Subject to biased selection of studies • Subject to biased interpretations

11. Goals of Explicit,Evidence-based Approach • Credibility • Transparency • People can understand what you did • Systematic • Reproducibility, limit bias • Different people would get same result • Identify gaps in evidence • Highlight where we need better evidence • Reduce the chance of “getting it wrong”

12. Explicit, Evidence-based Process Specifies: • Key questions to be answered • Consistent process for identifying and critically reviewing evidence • Procedures to reduce bias and conflict of interest

13. Dynamic Relationship Between Evidence Review & Synthesis and Evidence-based Decision Making EconomicInformation Framing Key Questions Rigor Required • Evidence Review • and Synthesis • (Formulary Committee Staff, • EPCs, DERP) Evidence-Based Decision Making (Formulary Committee of PBM, Private Payer, States) Budget Constraints Studies 1 2 Evidence Review Acceptability 3 Decisions Teutsch S, Berger M, Evidence Synthesis and Evidence-based Decision Making: Related But Distinct Processes. Medical Decision Making2005;25:487-9.

14. If we want more evidence-based practice, we need more practice-based evidence. Larry Green (www.lgreen.net)

15. Integrating Evidence into Practice and Research that Informs Real-World Practice

16. Integration of Evidence-Based Processes into Practice • Efficacy vs. Effectiveness • Decision oriented • Post-marketing surveillance • Quasi-experimental methods • Learning networks (voluntary, self-organizing) • Rapid cycle from problem identification to hypothesis testing • Integration of evidence into practice • Quality improvement and measurement

17. The Two Faces of “TRIP” • Funding research on transferring research into practice versus • Embedding the transfer of research into practice within the culture of universities, health care organizations and research funders

18. Efficacy vs. Effectiveness

19. Effectiveness Trials • Trials for which the hypothesis and study design are formulated based on information to make a decision. Tunis et al., 2003 • Focus is gathering information of maximum interest to clinicians and other decision-makers. March et al., 2005

20. Explanatory-Pragmatic Domains for Practical Clinical TrialsPRaCTIHC workgroup • Eligibility criteria • Flexibility of the intervention • Practitioner expertise • Follow-up intensity • Follow-up duration • Participant compliance • Practitioner adherence to protocol and intervention • Primary analysis scope and specification

21. Quality Improvement • Guidelines and standards based on evidence • System change to assure delivery of services • EHR • Disease management • Performance metrics and incentives

22. Adherence to Quality Indicators, Overall and by Type of Care and Function McGlynn E, et al. NEJM 2003

23. Translating Research into Practice Study systems for quality improvement • Organization and structure of care • Reimbursement and incentives • Information systems • Community and patient engagement • Training

24. Consequences of EBM Paradigm Basic Science Biomedical Model Clinical Research Comparative Effectiveness Inform R&D Needs Relative Value (Cost Effectiveness) EBM Literature Synthesis EBM / HECON Model Determine Appropriate Care Evidence-based Guidelines Policy Decisions Reimbursement, Coverage and Incentives Quality Improvement Determine Outcomes Of Care Determine Access to Services

25. Thanks! steven_teutsch@merck.com