1. 1 Comparative Effectiveness Research: Key Issues and Controversies Consumer-Purchaser Disclosure Project Discussion Forum May 5, 2009 Steven D. Pearson, MD, MSc, FRCP

2. 2 Background Policy givens: –Unsustainable cost increases –Unexplainable variation in practice patterns –Not enough evidence for decisions about new treatments International efforts (health technology assessment) –NICE in England “Comparative Effectiveness” –Stark bill –Baucus bill American Recovery and Reinvestment Act (ARRA) stimulus bill funding for Comparative Effectiveness Research (CER)

3. 3 10-Year Impact on Spending of a Center for Comparative Effectiveness Dollars in billions SAVINGS COSTS Source: Based on estimates by The Lewin Group for The Commonwealth Fund, 2007.

4. 4 Chief remaining questions on CER Stimulus spending –Priorities for spending at AHRQ and NIH –Secretary of HHS $400 million –Inclusion of cost and/or cost-effectiveness CER 2.0 –Structure –Governance –Funding –Priority Setting –Research Methods (cost-effectiveness) –Implementation

5. 5 Stimulus spending Priorities for spending at AHRQ and NIH –Mix of systematic reviews and prospective studies –Framing of topics as “drug vs. drug” or broader pathways of care –Studies of health plan policies such as prior authorization Secretary of HHS $400 million Inclusion of cost-effectiveness

6. 6 Weighing up costs and effects Cost ($) Effectiveness New treatment more effective, less costly New treatment less effective, more costly High extra cost Low gain Low extra cost High gain

7. 7 Why Costs? “Not to consider costs is delusional” Costs should be considered transparently and always in the context of clinical effectiveness Without consideration of cost No societal support for explicit cost considerations in clinical decisions and medical policies All explicit health plan efforts will be suspect Continued difficulty negotiating prices in relation to evidence of incremental benefit Marginal benefit at high price will continue to be a dominant market signal for manufacturers

8. 8 How to do Costs? Carve-out Commissioned by individual payers, including Medicare Arms’ length Funded as part of CER stream but function delegated to an allied yet separate organization Carve-in Distrust of clinical effectiveness judgments if mixed with costs More efficient to nest within same effort to generate a systematic review of the clinical evidence Benefits from the objectivity and transparency of a federal comparative effectiveness initiative to gain broad acceptance

9. 9 Legislation for CER 2.0 Structure Inside or attached to government vs. independent? Governance Stakeholders on the Governing Board or only on Advisory Committees? Funding How much from private health plans and purchasers? Priority Setting Who and how? Research Methods Cost-effectiveness yea or nea? Implementation

10. 10 http://www.politico.com/singletitlevideo.html?bcpi d=1155201977&bctid=21157881001

11. 11 How will CE information be used? Concerns –Limit access to life-saving treatments just because of cost “One-size-fits-all” methodologies and applications to coverage policies Cost-effectiveness applied as a strict cut-off for coverage Cost-effectiveness devalues older, sicker patients –Put governmental bureaucrats between you and your doctor –Stifle innovation

12. 12 How CER should be used “Too cold” –Dissemination of information to patients and clinicians “Too hot” –Direct mandates for “all-or-nothing” coverage decisions “Just right” –Providing “guidance” to patients, clinicians, and payers –Application by payers to create value-based tools and policies in support of optimal care and to ensure best use of every health care dollar Patient-clinician decision support Reimbursement policy Value-based insurance design Physician group compensation (P4P)

13. 13 Application of Cost-effectiveness 1.Help identify the least costly alternative among equivalent treatment options 2.Provide some context for the additional cost paid for very marginal clinical benefits 3.Help anchor initial pricing for new technologies in evidence of their marginal (if any) benefit Tools –Patient-clinician decision tools –Reimbursement policy –Value-based insurance design –Physician group compensation (P4P) to align incentives

14. 14 For further information: spearson@icer-review.org www.icer-review.org