Practical Clinical Trials and the AHRQ Experience

1. Practical Clinical Trials and the AHRQ Experience Carolyn M. Clancy, MD Director Agency for Healthcare Research and Quality AIFA European Conference on Clinical Research for Decision-Making Rome – March 30, 2007

2. AHRQ & PCTs • AHRQ Overview • Personalized Health Care • Practical Clinical Trials • Q&A

3. AHRQ’s Mission Improve the quality, safety, efficiency and effectiveness of health care for all Americans

4. HHS Organizational Focus NIH Biomedical research to prevent, diagnose and treat diseases CDC Population health and the role of community-based interventions to improve health AHRQ Long-term and system-wide improvement of health care quality and effectiveness

5. Combating the “Sting of Ignorance” “The nation faces two yawning medical information gaps. First, we need more studies comparing treatments… what about the insurers, private and governmental, who pay such a large share of the nation’s $220 billion annual drug bill? They could support such studies with the rounding error of their annual budgets — and then save billions if the findings were put Into practice. The second problem is that much of the knowledge we do have is not communicated…we need an unbiased, efficient system to get the word out to practitioners on what works best.” Jerry Avorn, Professor, Harvard Medical School New York Times Op-Ed September 16, 2006

6. Comparative Effectiveness:Effective Health Care Program • Designed to support the new Medicare prescription drug benefit in 2006 • Mandated by Section 1013 of the Medicare Modernization Act to improve the quality, effectiveness and efficiency of health care delivered through Medicare, Medicaid and the S-CHIP programs • Builds on years of experience gained through AHRQ’s Evidence-Based Practice Centers

7. Effective Health Care Program Translate Evidence into Improvements (Eisenberg Clinical Decisions & Communications Science Center) Review/Compare Existing Evidence (Evidence-Based Practice Centers) Accelerate Practical Studies (DEcIDE Network)

8. Comparative Effectiveness Review: Management Strategies for Renal Artery Stenosis • Major Finding: No clear advantage found to treating narrowed kidney arteries with angioplasty, with or without a stent, over drug therapy • Narrowed kidney arteries are most common cause of correctable high blood pressure • Drug therapy and angioplasty both improve blood pressure and slow down the worsening of kidney function AHRQ Comparative Effectiveness Review No.5, Comparative Effectiveness of Management of Renal Artery Stenosis, October 2006, Available at www.effectivehealthcare.ahrq.gov

9. Comparative Effectiveness Reviews in Progress • Comparative Effectiveness of Drug Therapies for Rheumatoid or Psoriatic Arthritis • Comparison of Therapies for Low Bone Density • Comparison of Therapies for Clinically Localized Prostate Cancer • Evidence of Off-Label Use of Atypical Anti-Psychotic Medications

10. Sample DEcIDE Projects

11. Eisenberg Clinical Decisions and Communications Center • The knowledge is used to maximize the benefits of health care, minimize harm and optimize the use of health care resources • Presents evidence synthesized by the Evidence-Based Practice Centers and generated by the DeCIDE Network in actionable formats for stakeholders including patients, providers, policymakers and the public Oregon Health & Sciences University

13. http://effectivehealthcare.ahrq.gov/dsc/products.cfm New Eisenberg Center Summary Guides

14. 2008 Priorities Patient Safety • Health IT • Patient SafetyOrganizations • New PatientSafety Grants Effective HealthCare Program AmbulatoryPatient Safety • Comparative Effectiveness Reports • Network of Research Centers • Clear Findings for Multiple Audiences • Safety & Quality Measures,Drug Management andPatient-Centered Care • Patient Safety ImprovementCorps Medical ExpenditurePanel Surveys Other Research & Dissemination Activities • Quality & Cost-Effectiveness, e.g.Prevention and PharmaceuticalOutcomes • U.S. Preventive ServicesTask Force • Visit-Level Information on Medical Expenditures • Annual Quality & Disparities Reports

15. Questions are the Answer • Patient Involvement Campaign by AHRQ and the AD Council • Series of TV, radio and prints Public Service Announcements • A Web site that features a “Question Builder” for patients to enhance their medical appointments • The message: Get More Involved With Your Health Care

16. FY 2008 Request • FY 2008 Request = $329,564,000 • Increase of $10,872,000, or 3.4%, from the FY 2007 Continuing Resolution Level • Department-wide $15,000,000 Initiative related to Personalized Health Care

17. FY 2008 Initiative:Personalized Health Care • Personalized Health Care • +$15 million • The initiative will accelerate the movement toward personalized health care and help bring “next generation” effectiveness of care for individual patients. • This initiative is essential to our drive for health care transparency by identifying and consistently measuring effective, high quality care.

18. Partnership ELECTRONIC MEDICAL RECORD CLINICAL RESEARCH CLINICAL RESEARCH CLINICAL RESEARCH CLINICAL RESEARCH PUBLIC-PRIVATE PARTNERSHIP

19. Disease Diagnosis Gene Therapy Drug Design The Promise of Genomics “We stand on the threshold of creating a future that will revolutionize the practice of medicine by allowing us to predict disease, develop more precise therapies and, ultimately, pre-empt the development of disease in the first place.” Elias Zerhouni, 2006

20. Specialized Care Examples of interventions/diagnostics that can help specific groups of people Iressa – Foradvanced non-small cell lung cancer treatment. A gene-based test can help identify patients who might be helped by the drug Gleevec – For treatment of chronic myelogenous leukemia (gene-based)

21. Challenges • Using data on outcomes of gene-based testing, which currently is rarely collected • Acquiring data on diagnostics, therapeutics and patient outcomes from many different databases • Using administrative data for research purposes • Securely linking data from multiple resources

22. Solutions • A secure Web-based system for sharing gene-based data among multiple organizations • Partnerships that encourage research, development and increased implementations • Shared decision-making

23. Are patient outcomes improved? What are the harms? Do the benefits outweigh the harms? What is the incremental benefit? Is the real-world net benefit the same as in clinical trials? What are the costs? Given the net benefit and costs, is the intervention justified? Key Questions for Decision-Makers

24. Reliable evidence is essential to improving health care quality and to supporting efficient use of limited resources

25. Question Hypothesis Study Publications Changes in practice Clinical Evaluation Science: Phase 1

26. Clinical Evaluation Science: Phase 2 Publications (multiple) Evidence synthesis Guidelines, Performance Measures Changes in Practice

27. Practical Clinical Trials • Compare clinically relevant interventions • Enroll a diverse study population • Recruit from a variety of practice settings • Measure a broad range of relevant health outcomes

28. PCTs vs. ECTs

29. AHRQ GOLD Grant: Cluster Randomized Trials • HMO research Network CERT – Harvard Pilgrim Health Care, Wellesley, MA • CRTs are rarely used to study the comparative effectiveness of multiple treatments, such as two different drug regimens. This study: • Issues that must be addressed in order to perform CRTs in a manner that is acceptable to all parties, feasible and methodologically sound • Evaluate medical ethics and develop ethical standards to inform researchers and health plans considering CRTs. Duration of Project September 1, 2000 – September 29, 2007

30. Istituto di Ricerche Farmacologiche Mario Negri • 2001 study of 3,567 outpatients with type 2 diabetes, using AHRQ’s Diabetes PORT • Findings involving101 outpatient diabetic clinics and 103 general practitioners throughout Italy: • Self-monitoring of blood glucose can improve metabolic control as part of a strategy for promoting patient autonomy • For patients not treated with insulin, self-monitoring is associated with higher blood glucose levels and psychological burden Istituto di Ricerche Farmacologiche Mario Negri

31. The Guide to Clinical Preventive Services 2006 • Contains 53 new or revised clinical preventive services recommendations from the U.S. Preventive Services Task Force • AHRQ and United Health Foundation to distribute 430,000 copies to primary care physicians

32. Electronic Preventive Services Selector (ePSS) • A quick hands-on tool designed to help primary care clinicians identify screening, counseling and preventive medication services appropriate for patients • PDA and Web versions based on current USPSTF recommendations, searchable by specific patient characteristics • Available at www.ePSS.ahrq.gov Consumer Version Underway

33. Why is the Supply of PCTs Inadequate? • Inadequate public and private funding • Costly and often longitudinal studies • Lack of organized mechanism to identify high priority questions for decision makers

34. Increasing PCTs Supplies • Development of a mechanism to establish priorities for these studies • Significant expansion of an infrastructure to conduct clinical research within the health care delivery system • More reliance on high-quality evidence by health care decision makers • Substantial increase in public and private funding for these studies

35. Strategies for Improvement • Systematically identify and prioritize knowledge gaps • Decision makers Insist on high-quality evidence in making decisions • Create operational infrastructure • Address Methodological and Ethical Issues • Examine funding options

36. Your questions?