1. 1 Evidence and the next stage of health care reform: Why consumer engagement is so important Steven D. Pearson, MD, MSc President, Institute for Clinical and Economic Review

2. What do we mean by “evidence?” “Evidence” can have a bad aura among consumers and consumer advocates –Population-based instead of about the individual –Applied by insurers in ways that restrict access to tests and treatments desired by patients Evidence-based medicine is the conscientious, explicit and judicious use of current best evidence in making decisions about the care of individual patients. –Sackett, 1996 2

3. Why better evidence became part of health care reform Much of what doctors do is not based on good evidence –Does FDA approval mean there is good evidence? –Lack of good evidence commonly leads to inappropriate and often unnecessary treatment Autologous bone marrow transplant for metastatic breast cancer Generic drugs for rheumatoid arthritis found to be as good as more expensive and risky new drugs “Tens of thousands of women each year might be able to skip at least some of the grueling treatments for breast cancer — which can include surgery, heavy chemo and radiation — without greatly harming their odds of survival.” Choosing Wisely – will be discussed by Dr. Santa 3

4. Evidence-based medicine and health care reform Comparative effectiveness research (CER) –Research comparing alternative care options to determine what works best for whom, under real- world conditions Systematic review of existing published evidence Generation of new evidence through clinical trials but also clinical registries and existing database analyses Patient-centered outcomes research (PCOR) –PCORI Health care reform did not focus on the evidence comparing doctors and hospitals 4

5. Why consumer engagement can be so powerful Helping to identify research topics –PCORI –Things over which patients have control, e.g. diet, exercise Helping to identify outcomes that are more patient-centered –Medications for pediatric seizures Helping to interpret and guide the application of results –Examples from CEPAC and CTAF 5

7. CEPAC and CTAF Collaboration among –Patient groups Connecticut Center for Patient Safety Maine Quality Counts Center for Healthcare Decisions Patient Advocates in Research –State Medicaid programs –Private plans –Large physician-hospital groups Independent Council meets in public to discuss tailored evidence reviews on effectiveness and cost, to vote on evidence, and to make recommendations for best practice and policy All stakeholders work together afterward to implement the findings 7

8. New England CEPAC: Topics and Impact Treatments for Attention-Deficit Hyperactivity Disorder (ADHD) –Helped interpret evidence and disseminate to patient networks in order to shift to behavioral therapy for youngest children Treatment for severe depression –Perspectives on severity of condition and impact on families part of deliberation that led to first-in- nation coverage for new magnetic treatment Supplemental screening options for women with dense breast tissue 8

10. Supplemental screening for women with dense breast tissue Consumer engagement critical in –Perspective on importance of the concern for masking as opposed to breast cancer risk –Judgments of the relative balance of benefits of identifying additional cancers and the harms of false positive results, biopsies, and anxiety –Determination of the information patients want and how to present it to women (see handout) –Dissemination of the report and materials to patient groups throughout California, New England, and nationally 10

11. Conclusion Evidence, its generation, interpretation, and application to improve patient outcomes and the patient experience, is an important component of the next phase of health care reform. Consumers have a critical role to play in advancing the best aspects of evidence-based medicine in the interests of patients now and in the future. There is a shift occurring among clinicians and researchers that offers a new opportunity for engagement. Seize it! 11

12. 12 Thank you Steve Pearson spearson@icer-review.org