Comparative Effectiveness Research: Relevance and Applications to Pharmacy

1. Comparative Effectiveness Research: Relevance and Applications to Pharmacy This activity was developed by the American Pharmacists Association and the Agency for Healthcare Research and Quality.

2. Speaker Two Glen T. Schumock, Pharm.D., M.B.A. Director of the Center for Pharmacoeconomic Research Professor of Pharmacy Practice, the University of Illinois at Chicago Speaker Three A. Simon Pickard, B.Sc.Pharm., Ph.D. Assistant Director of the Center for Pharmacoeconomic Research Associate Professor of Pharmacy Practice, the University of Illinois at Chicago Moderator Carmen Kelly, Pharm.D., R.Ph. Pharmacist Officer Agency for Healthcare Research and Quality Effective Health Care Speaker One Scott Smith, R.Ph., M.S.P.H., Ph.D. Director of Pharmaceutical Outcomes Research Agency for Healthcare Research and Quality Effective Health Care Speakers

3. APhA is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. The ACPE Universal Activity Number assigned to the activity by the accredited provider is: 202-999-09-224-H04-P. This activity is approved for 1 contact hour of continuing pharmacy education credit (0.1 CEU). To obtain continuing pharmacy education credit for this activity, participants must participate in the entire activity, and complete the online activity evaluation form located on www.pharmacist.com/education by September 23, 2009. A Statement of Credit will be automatically generated upon achieving these requirements. There is no fee to participate in this activity. ACPE Activity Type: Knowledge-Based Initial Release Date: September 9, 2009 Accreditation

4. Disclosures Glen T. Schumock, Pharm.D., M.B.A. has no financial interests or relationships to disclose. A. Simon Pickard, B.Sc.Pharm., Ph.D. has no financial interests or relationships to disclose. Scott Smith, R.Ph., M.S.P.H., Ph.D., and Carmen Kelly, Pharm.D, R.Ph.,have no financial interests or relationships to disclose. AHRQ’s Office of Communications and Knowledge Transfer staff, The Lewin Group project staff, and APhA’s editorial staff declare no conflicts of interest or financial interests in any product or service mentioned in this program, including grants, employment, gifts, stock holdings, and honoraria.

5. Disclaimer This presentation and slide set do not represent the policy of either the Agency for Healthcare Research and Quality (AHRQ) or the U.S. Department of Health and Human Services (DHHS). The views expressed herein are those of the presenter, and no official endorsement by AHRQ or DHHS is intended or should be inferred. Current information about the Effective Health Care Program should be obtained from AHRQ, and not from these slides.

6. Learning Objectives • Describe AHRQ’s Comparative Effectiveness Program and provide an overview of the factors and events leading to the current national interest in comparative effectiveness • Identify scientific evidence-based resources, including clinician and patient guides, produced through AHRQ’s Comparative Effectiveness Program that are relevant for use in their practice • Understand the process for submitting topics to AHRQ for future comparative effectiveness research • Compare the differences between comparative effectiveness studies and traditional efficacy studies • Explain the relevance of comparative effectiveness research to pharmacy practice

7. Comparative Effectiveness Research and AHRQ’s Effective Health Care Program Scott Smith, R.Ph., M.S.P.H., Ph.D. Agency for Healthcare Research and Quality Effective Health Care

8. CBO Comparative Effectiveness Definition Comparative Effectiveness “…a rigorous evaluation of the impact of different options that are available for treating a given medical condition for a particular set of patients.” Congressional Budget Office, 2007

9. Effective Health Care (EHC) Program, 2003 – Present • Authorized in 2003 by Section 1013 of the Medicare Prescription Drug, Improvement, and Modernization Act • Conducts objective comparisons of the effectiveness of different health care interventions • Goal: To support informed health care decisions by patients, clinicians, and policymakers and improve the quality, effectiveness, and efficiency of health care to support evidence-based practice

10. Effective Health Care Program • To improve the quality, effectiveness, and efficiency of health care delivered through Medicare, Medicaid, and CHIP programs • Focus is on what is known now: ensuring programs benefit from past investments in research and what research gaps are critical to fill • Focus is on clinical effectiveness

11. Synthesizes existing scientific evidence Generates new scientific evidence to address gaps Translates research into plain-language guides Approaches to Research

12. Effective Health Care Program

13. Available EHC Products

14. Clinician Resources • Concise • Actionable • Paired with consumer guides • Convey level of uncertainty/certainty of findings

15. Arthritis and non-traumatic joint disorders Cancer Cardiovascular disease, including stroke and hypertension Dementia, including Alzheimer’s Disease Depression and other mental health disorders Developmental delays, attention-deficit hyperactivity disorder, and autism Diabetes mellitus Functional limitations and disability Infectious diseases, including HIV/AIDS Obesity Peptic ulcer disease and dyspepsia Pregnancy, including pre-term birth Pulmonary disease/asthma Substance abuse Priority Conditions for the Effective Health Care Program

16. Comparative Effectiveness Reviews: New and Coming Soon… • Comparative Effectiveness Reviews • Stable Ischemic Heart Disease (draft report) • Particle Beam Therapies for Cancer(technical brief) • Stereotactic Radiosurgery (technical brief) • Heart Valve Replacement (technical brief) • Core Needle Breast Biopsy • Lipid-Modifying Agents • Radiofrequency Catheter Ablation for Atrial Fibrillation • Chemotherapy Agents in the Prevention of Breast Cancer • 9 Comparative Effectiveness Review Updates

17. Summary Guides: New and Coming Soon… • Summary guides - Osteoarthritis of the Knee (consumer and clinician) - Gestational Diabetes (consumer and clinician) - Induction of Labor (consumer and clinician) - Particle Beam (policymaker) • Spanish translations are available for 9 summary guides

18. Coming Soon… (cont.) Interactive Decision Aids

19. Important Role of Outside Input • Nomination of research topics • Input on research questions • Comment on draft reports • Focus test translation products • Comments on overall program direction and quality improvement

20. Topic Nomination Form http://effectivehealthcare.ahrq.gov/aboutUs.cfm?abouttype=program#Topic

21. Selection Criteria Appropriateness • 3 criteria • 7 criteria • 1 criterion • 1 criterion • 6 criteria Importance Duplication Feasibility PotentialImpact

22. Transparency • Nominations are posted on the Web site • Research selection process including selection criteria are posted on the Web site • Moving toward posting all of the final decisions on the Web site

23. How To Stay Informed • EHC Web Site • E-Mail Notices Through Listserv • Select to receive information on a range of topics: • Arthritis and nontraumatic joint disorders • Cancer • Cardiovascular disease, including stroke and hypertension • Dementia, including Alzheimer's Disease • Depression and other mental health disorders • Developmental delays, attention-deficit hyperactivity disorder, and autism • Diabetes mellitus • Functional limitations and disability • Infectious diseases, including HIV/AIDS • Obesity • Peptic ulcer disease and dyspepsia • Pregnancy, including preterm birth • Pulmonary disease/asthma • Substance abuse

24. Are you currently using, or planning to use, AHRQ's Comparative Effectiveness reports in your setting? Yes, currently Yes, within the next 12 months Yes, after 12 months or later No Polling Question

25. Comparative Effectiveness Research: Relevance and Applications to Pharmacy Glen T. Schumock, Pharm.D., M.B.A. A. Simon Pickard, B.Sc.Pharm., Ph.D. University of Illinois at Chicago

26. Health and Health Care in the United States • The U.S. continues to spend more on health care, including prescriptions, than other countries • Outcomes of the health care system are not better in the U.S. compared to other developed countries • There is much variation in the provision and cost of care between regions of the US

27. Health Care Spending Per Capita Source: Congressional Research Service. Washington, DC. Pub No. RL34175. Based on 2003 data from the Organisation for Economic Co-operation and Development (OECD)

28. Pharmaceutical Spending Per Capita Source: Congressional Research Service. Washington, DC. Pub No. RL34175. Based on OECD data 2006.

29. Global Trends in Health Expenditures From: http://www.oecd.org/document/16/0,3343,en_2649_34631_2085200_1_1_1_1,00.html

30. Health Care Spending Per Capita and Life Expectancy Source: Congressional Research Service. Washington, DC. Pub No. RL34175. Based on OECD data 2006

31. Health Care Spending and Self-Reported Health Status Source: Congressional Research Service. Washington, DC. Pub No. RL34175. Based on OECD data 2006

32. Projected Federal Spending

33. Per Capita Medicare Spending: Regional Variations From: Congressional Budget Office. Research on Comparative Effectiveness of Medical Treatments. 2008.

34. The Outcomes Movement • Geographic variation in practice patterns • Poor relationship between costs and outcomes • Need to establish best practices • Cost containment • Recognition of limited resources • System management • Improved management, accountability A. Epstein, NEJM 1990

35. Information Gap • Many gaps in the literature • Need evidence to support rational decisions in health care • Appropriateness • Patients of interest/indication • Comparators/therapeutic alternatives • Study design • Outcomes measured • The drug development and approval process in the U.S. does not provide evidence necessary to clinicians or policymakers to choose between drugs for the same indication

36. Effective Health Care • Address variations in practice patterns by adopting best practices • Address evidence gaps in best practices by generating evidence using appropriately designed studies • Secondary effect of best practices is cost containment (not directly addressed in legislation) • Improve the management of health care systems and accountability of providers through measuring and monitoring outcomes

37. Definitions of Comparative Effectiveness Research (CER) Comparative effectiveness research is: • The conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat, and monitor health conditions in “real world” settings. • To improve health outcomes by developing and disseminating evidence-based information to patients, clinicians, and other decisionmakers, responding to their expressed needs, about which interventions are most effective for which patients under specific circumstances. • 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.

38. Purpose of Comparative Effectiveness Research (CER) The purpose of CER is: • to assist consumers, clinicians, purchasers, and policymakers to make informed decisions that will improve health care at both the individual and population level. (Federal Coordinating Council 2009) (IOM 2009)

39. Two Essential Elements • Comparison of two or more agents or interventions that are considered true therapeutic alternatives • Examination of the effects/outcomes in actual practice (i.e., effectiveness) • U.S.-based initiatives have framed outcomes/effects in terms of benefits/harms • More broadly, they include economic outcomes as well as clinical and humanistic benefits/harms

40. Types of Comparative Effectiveness Research • Primary comparative effectiveness • Retrospective observational studies (case control or cohort studies) • Prospective observational studies (aka “large simple clinical trial,” “pragmatic clinical trials”) • Cluster randomized studies • Registry-based studies • Secondary comparative effectiveness • Systematic review and meta-analyses • Modeling and decision-analysis

41. Differences Between Efficacy and Effectiveness Studies Source: Schumock & Pickard, AJHP 2009

42. Example: CER (primary retrospective) • Patients: • Adults with heart failure • Intervention: • Drug therapy (beta-blockers) • Comparators: • Atenolol, metoprolol, carvedilol, others • Endpoint/outcome • Death

43. Example: CER (primary prospective) • Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) • Patients: • Schizophrenia • Intervention: • Drug treatment (antipsychotics) • Comparators: • Olanzapine, perphenazine, quetiapine, risperidone, ziprasidone • Endpoint/outcome • Treatment failure (time to discontinue)

44. Example: CER (secondary – meta-analysis) • Patients: • Chronic obstructive pulmonary disease (COPD) • Intervention: • Drug therapy (anticholinergics) • Comparators: • Ipratropium or tiotropium vs. control (placebo or active comparator) • Endpoint/outcome • Death • Myocardial infarction (MI) • Stroke

45. Applications of Comparative Effectiveness Research • CER data can be used to help inform: • Individual patient care decisions/recommendations (patient-level) • Policy such as formulary decisions (system-level) • CER data can add to body of evidence on: • Effectiveness of one drug compared to another • Safety of one drug compared to another • Outcomes from CER studies provide inputs for cost-effectiveness analyses and decisions

46. The 3T’s Road Map Dougherty D, Conway PH. High-Quality Care: The "3T’s" Road Map to Transform US Health Care: the "how" of high-quality care. JAMA. 2008;299(19):2319-2321

47. CER and the Role of the Pharmacist • Understand study design and methods used in comparative effectiveness research • Understand differences between efficacy and effectiveness studies and their strengths and weaknesses • Monitor literature for results of comparative effectiveness studies • Integrate evidence from these studies with existing knowledge base relevant to patient and system-level decisionmaking

48. Will Comparative Effectiveness (CE) Affect Pharmacy Practice? • Scientific considerations issues: • Timeliness of evidence • Rigor and quality of methods • Ability to widely disseminate results of studies • Policy-based initiatives issues: • Uncertainty about health care reform • Extent to which medications are a priority area for CER • Whether CE is eventually required for regulatory approval • Cost as an explicit criteria by which to evaluate alternatives • If costs are not considered, how is value for money to be assessed? • Clinical practitioners and professional societies’ issues: • How well CE evidence is understood and disseminated • Extent to which CE evidence supports or does not support current practice • Willingness and ability of clinicians to change practice • Reimbursement structure may not incentivize providers to act upon evidence

49. Final Thoughts on Relevance • CE-based evidence is relevant to all stakeholders in health care • Physicians, pharmacists, health care provider organizations, pharmaceutical manufacturers, employers, insurers, government agencies • CE is not the only solution needed to address excessive health care costs in the US, but it can be an important part of the solution • While CE is not limited to pharmaceuticals, these are often cited as the quintessential example of where CE is needed

50. For More Information See: Am J Health-System Pharm 2009;66:1278-1286.