1. Evidence, HTA and Comparative Effectiveness in the U.S. Presentation at AMCP March 28, 2007 Peter J. Neumann Tufts-New England Medical Center

2. Overview HTA around the world HTA around the world American exceptionalism? American exceptionalism? A new U.S. Center for Comparative Effectiveness? A new U.S. Center for Comparative Effectiveness? Looking ahead (implications for VA) Looking ahead (implications for VA)

3. HTA Around the World

4. ISPOR MEMBERSHIP BY REGION 2006

5. ISPOR Chapters

6. American Exceptionalism?

8. Explaining American exceptionalism in health care The values argument The values argument The “system” argument The “system” argument The political obstructionist argument The political obstructionist argument

9. The U.S. Landscape Medicare Medicare AHRQ AHRQ Medicaid (the DERP) Medicaid (the DERP) Regional efforts Regional efforts Other (VA!) Other (VA!)

10. Medicare spending trends Source: 2006 Medicare Trustees Report Total Part A Part B Part D Medicare Spending Per Beneficiary

11. A New Center for Comparative Effectiveness?

13. Activities of a Comparative Effectiveness Center Conducting and/or sponsoring research Conducting and/or sponsoring research Setting research priorities Setting research priorities Providing a forum for methodological and other issues Providing a forum for methodological and other issues

14. Placement Options Within CMS Within CMS Within AHRQ Within AHRQ Within NIH Within NIH Within FDA Within FDA Creation of a new government agency Creation of a new government agency Creation of a quasi-public organization Creation of a quasi-public organization

15. Governance, Oversight, and Funding Institutional independence Institutional independence Separation of assessment from decision making Separation of assessment from decision making Funding Funding

16. Substantive Issues Methodological rigor Methodological rigor Analytic perspective Analytic perspective Objectivity and independence Objectivity and independence Transparency Transparency Stakeholder input Stakeholder input Appeals process Appeals process Identifying research priorities Identifying research priorities Updating conclusions over time Updating conclusions over time

17. Implications For the collection of evidence For the collection of evidence For patient access For patient access For innovation For innovation For patient health For patient health

18. Key issues Level of evidence required Level of evidence required Tradeoff between rigor and timeliness Tradeoff between rigor and timeliness The process for evaluation and decision making The process for evaluation and decision making Role of CEA in HTA Role of CEA in HTA

19. Will there be a role for cost- effectiveness?

20. NICE in America?

21. NICE Decisions by Cost-Effectiveness Threshold (1999-2003) < 20 20-30 >30 20-30 < 20

22. Selected cost-effectiveness ratios for technologies covered by Medicare Implantable cardioverter defibrillators: $30,000- $85,000/QALY Implantable cardioverter defibrillators: $30,000- $85,000/QALY Lung-volume reduction surgery: $98,000- $330,000/QALY Lung-volume reduction surgery: $98,000- $330,000/QALY Left-ventricular assist devices: $500,000-$1.4 million/QALY Left-ventricular assist devices: $500,000-$1.4 million/QALY PET for Alzheimer’s disease (Over $500,000) PET for Alzheimer’s disease (Over $500,000) Source: Neumann et al., 2005

24. Why don’t Americans use CEA? Conflicting/weak incentives Conflicting/weak incentives Quality of evidence is problematic Quality of evidence is problematic Regulatory/legal barriers Regulatory/legal barriers Ethical concerns Ethical concerns Lack of infrastructure Lack of infrastructure Lack of cultural acceptance Lack of cultural acceptance

25. The role of the AMCP Format

26. An audit of 115 dossier, 2002-2005 Characteristics Total # of Observations % Positive result (n) Statement on form of economic analysis (even if wrong) 106 59% (62) Statement of viewpoint of analysis 106 38% (40) Time horizon for costs and benefits stated 106 78% (83) Discounting if analysis 2 years or longer 44 34% (15) All assumptions are clearly stated 106 20% (21) Compared product to all relevant comparators 106 37% (39)

27. Audit (page 2) Characteristics Total # of Obs. % Positive results (n) Reports sensitivity analysis performed 106 43% (46) All conclusions follow from data reported 106 54% (57) Conclusions accompanied by specific caveats 106 18% (19) Report mentions that comparators might be superior given changes in assumptions 106 8% (8)

28. Six trends to watch… Rise of HTA organizations and evidence requirements Rise of HTA organizations and evidence requirements Increasing parsing of clinical evidence Increasing parsing of clinical evidence Coverage with evidence development Coverage with evidence development Cost-effectiveness requirements Cost-effectiveness requirements Flexible evidentiary standards Flexible evidentiary standards  Quantification of risks/benefits  Adaptive clinical trials Linking evidence to payment Linking evidence to payment