1. The Latest Federal Initiatives in Evidence-Based Medicine Barry M. Straube, M.D. Centers for Medicare & Medicaid Services Citizen’s Council on Health Care November 10, 2005

2. 2 Centers for Medicare & Medicaid Services (CMS) Provides health benefits for over 76 million Americans Provides health benefits for over 76 million Americans Medicare Medicare Medicaid Medicaid State Children's Health Insurance Program (SCHIP) State Children's Health Insurance Program (SCHIP) Spends over $600 billion annually for health care services in CMS programs Spends over $600 billion annually for health care services in CMS programs

3. 3 Centers for Medicare & Medicaid Services (CMS) Healthcare Benefits Administration Healthcare Benefits Administration Establish payment methodology for providers Establish payment methodology for providers Conduct research on financing, treatment & management Conduct research on financing, treatment & management Assure that contractors & state agencies run CMS programs correctly Assure that contractors & state agencies run CMS programs correctly Identify fraud & abuse, take appropriate action Identify fraud & abuse, take appropriate action Beneficiary Focused Activities Beneficiary Focused Activities Benefits and health education Benefits and health education Healthcare data for choice Healthcare data for choice Advocacy: Appeals, Grievances, patient rights Advocacy: Appeals, Grievances, patient rights Preventive services Preventive services

4. 4 Centers for Medicare & Medicaid Services (CMS) Quality-Focused Activities Quality-Focused Activities Laboratory Testing (CLIA) Laboratory Testing (CLIA) Survey & Certification of Health Care Facilities (LTC, SNF, HHA, Hospitals) Survey & Certification of Health Care Facilities (LTC, SNF, HHA, Hospitals) End-Stage Renal Disease Networks End-Stage Renal Disease Networks Organ Procurement Organizations Organ Procurement Organizations Quality Improvement Organizations Quality Improvement Organizations Multiple Demonstration Projects Multiple Demonstration Projects Health Information Technology promotion/adoption Health Information Technology promotion/adoption Pay-for-Performance, Paying for Quality, Value-Based Purchasing Pay-for-Performance, Paying for Quality, Value-Based Purchasing Healthcare data, public reporting Healthcare data, public reporting Evidence-Based Medicine, Technology & Innovation Evidence-Based Medicine, Technology & Innovation

7. A Variation Problem Dartmouth Atlas of Healthcare

10. 10 CMS Quality Roadmap VISION: The right care for every person every time VISION: The right care for every person every time Make care: Make care: Safe Safe Effective Effective Efficient Efficient Patient-centered Patient-centered Timely Timely Equitable Equitable

11. 11 CMS Quality Roadmap: Strategies 1. Work through partnerships to achieve specific quality goals 2. Publish quality measurements and information as a basis for supporting more effective quality improvement efforts 3. Pay in a way that expresses our commitment to quality, and that helps providers and patients to take steps to improve health and avoid unnecessary costs

12. 12 CMS Quality Roadmap: Strategies for QI 4. Assist practitioners in making care more effective and less costly, especially by promoting the adoption of HIT 5. Bring effective new treatments to patients more rapidly and help develop better evidence so that doctors and patients can use medical technologies and treatments more effectively

13. 13 Centers for Medicare & Medicaid Services (CMS) CMS as a Public Health Agency Using CMS influence and financial leverage to transform American healthcare system Using CMS influence and financial leverage to transform American healthcare system Quality, Value, Efficiency, Cost-effectiveness Quality, Value, Efficiency, Cost-effectiveness Assisting patients and providers in receiving evidence-based, technologically-advanced care Assisting patients and providers in receiving evidence-based, technologically-advanced care

14. 14 D.L. Sackett et al: BMJ 312, no.7023 (1996):71-72 Evidence-Based Medicine “Evidence-based medicine is the conscientious, explicit, and judicious use of current best evidence in making decisions about the care of individual patients.”

15. 15 D.L. Sacket et al, BMJ 312, no.7023 (1996): 71-72 Evidence-Based Medicine “The practice of evidence-based medicine means integrating individual clinical expertise with the best available external clinical evidence from systematic research and our patient’s unique value and circumstances.”

16. 16 Evidence-Based Medicine Evidence-Based Guidelines (EBGs) Evidence-Based Guidelines (EBGs) Different methods for designing guidelines Different methods for designing guidelines Global subjective judgment Global subjective judgment Consensus-based Consensus-based Evidence-based Evidence-based Outcomes-based Outcomes-based Preference-based Preference-based Guidelines should be based on evidence, not subjective judgment or opinion Guidelines should be based on evidence, not subjective judgment or opinion “Evidence-based”: coverage, performance measures, medical necessity, benefit design, disease management, quality improvement, regulations, & public policies “Evidence-based”: coverage, performance measures, medical necessity, benefit design, disease management, quality improvement, regulations, & public policies

17. 17 Features of EBGs Work of analyzing evidence and developing a guideline or policy is done by a small group of experts, usually sponsored by an organization Work of analyzing evidence and developing a guideline or policy is done by a small group of experts, usually sponsored by an organization Utilization of an explicit, rigorous process Utilization of an explicit, rigorous process End “product” is generic, applying to a class or group of patients, not individual patients End “product” is generic, applying to a class or group of patients, not individual patients Effects on care are indirect Effects on care are indirect Enable, motivate or (sometimes) force providers to deliver certain types of care to groups of people, NOT to a particular patient Enable, motivate or (sometimes) force providers to deliver certain types of care to groups of people, NOT to a particular patient

18. 18 Evidence-Based Medicine Evidence-Based Individual Decision Making (EBID) Evidence-Based Individual Decision Making (EBID) Focus on educating clinicians to help them bring more research and evidence into individual decisions about individual patients Focus on educating clinicians to help them bring more research and evidence into individual decisions about individual patients Integrating EBGs with EBID Integrating EBGs with EBID EBM should not just focus on individual physicians and their decisions EBM should not just focus on individual physicians and their decisions EBM is a set of principles and methods intended to ensure that to the greatest extent possible, medical decisions, guidelines, and other types of policies are based on and consistent with good evidence of effectiveness and benefit. EBM is a set of principles and methods intended to ensure that to the greatest extent possible, medical decisions, guidelines, and other types of policies are based on and consistent with good evidence of effectiveness and benefit.

19. 19 CMS Evidence-Based Initiatives National Coverage Decisions National Coverage Decisions Coverage with Evidence Development Coverage with Evidence Development Medicare Benefit Categories and specific benefits Medicare Benefit Categories and specific benefits Preventive Care Services Preventive Care Services Technology & Innovation Assessments Technology & Innovation Assessments Medical Devices Medical Devices Therapeutics Therapeutics Diagnostics Diagnostics Health Information Technology Health Information Technology Data collection via EHRs, registries, eprescribing, etc. Data collection via EHRs, registries, eprescribing, etc. Clinical Decision Support Clinical Decision Support

20. 20 CMS Evidence-Based Initiatives Quality & Efficiency Measure development Quality & Efficiency Measure development Quality Alliances Quality Alliances National Quality Forum National Quality Forum Constant reassessment Constant reassessment Drug Formulary for Medicare Prescription Drug Benefit Drug Formulary for Medicare Prescription Drug Benefit Pay-for-Performance Initiatives Pay-for-Performance Initiatives Hospital Hospital Physician Physician Home Health, Skilled Nursing Facilities, ESRD facilities Home Health, Skilled Nursing Facilities, ESRD facilities

21. 21 CMS EBM Initiatives QIO and ESRD Network Quality Improvement Activities QIO and ESRD Network Quality Improvement Activities CMS Breakthrough Initiatives CMS Breakthrough Initiatives Fistula First Fistula First Surgical Care Improvement Program (SCIP) Surgical Care Improvement Program (SCIP) Nursing Home Initiatives Nursing Home Initiatives Restraint reduction Restraint reduction Pressure ulcers reduction Pressure ulcers reduction Pain control Pain control Staff turnover rate reduction Staff turnover rate reduction

22. 22 Steps to Coverage Determination and Payment Outside of CMS: Congress determines benefit categories Congress determines benefit categories FDA approves drugs/devices for market FDA approves drugs/devices for market Within CMS: Benefit Category Determination Benefit Category Determination Coverage Decision Coverage Decision Coding Assignment Coding Assignment Payment Determination Payment Determination

23. 23 National Coverage Determinations (NCDs) Evidence-based decisions on whether to add or revise coverage for an item or service under Medicare Part A or B Must meet statutory “reasonable and necessary” standard Must meet statutory “reasonable and necessary” standard Juxtaposed to FDA “safe and effective” standards Juxtaposed to FDA “safe and effective” standards Binding on all contractors (supersedes any local coverage policies) Binding on all contractors (supersedes any local coverage policies)

24. 24 Most Coverage is Local Local 90% National 10% Local National

25. 25 What prompts NCDs? Internal (20%) or external (80%) requests Internal (20%) or external (80%) requests May involve: May involve: New technology or treatment not addressed in past New technology or treatment not addressed in past Reconsideration of a prior non-coverage (or limited coverage) decision Reconsideration of a prior non-coverage (or limited coverage) decision Response to significant variation in local coverage policies Response to significant variation in local coverage policies

26. 26 Key Factors Considered in National Coverage Determinations Must be potentially a benefit of Medicare Must be potentially a benefit of Medicare Evidence of improved health outcomes Evidence of improved health outcomes Appropriate for Medicare population Appropriate for Medicare population Replicable in provider community Replicable in provider community Costs not a primary factor historically Costs not a primary factor historically

27. 27 Recent NCD Guidance Documents CMS requested public comment on factors to consider in: Evidence development methods Evidence development methods Process for study design and implementation Process for study design and implementation Registries Registries Clinical trials Clinical trials Other methods Other methods

28. 28 Coverage with Evidence Development (CED) Used for promising innovations with insufficient evidence for individual patients or the Medicare population Used for promising innovations with insufficient evidence for individual patients or the Medicare population Also used when conclusive evidence is not available, but existing evidence strongly suggests probable benefit Also used when conclusive evidence is not available, but existing evidence strongly suggests probable benefit Offers prompt coverage linked with more evidence development Offers prompt coverage linked with more evidence development Speeds access, safeguards patients, improves evidence for better decisions Speeds access, safeguards patients, improves evidence for better decisions

29. 29 Coverage With Evidence Development = an alternative to non-coverage Is existing evidence sufficient to support Medicare coverage? Coverage Yes No Coverage with Evidence Development Non-coverage

30. 30 Flexible Coverage Processes Examples Prophylactic implantable cardioverter defibrillator (ICD) Prophylactic implantable cardioverter defibrillator (ICD) Data submitted to national registries, at low cost for participating hospitals all over the country Data submitted to national registries, at low cost for participating hospitals all over the country Otherwise would have more limited coverage Otherwise would have more limited coverage Expanded coverage to reach more patients Expanded coverage to reach more patients

31. 31 Flexible Coverage Processes Examples Additional off-label uses of cancer drugs Additional off-label uses of cancer drugs No FDA-approved results, and no studies covered in medical references No FDA-approved results, and no studies covered in medical references Previously would not have been nationally covered Previously would not have been nationally covered Evidence developed through clinical trials Evidence developed through clinical trials FDG-PET scanning FDG-PET scanning For dementia and neurodegenerative disorders For dementia and neurodegenerative disorders For cancer diagnosis, staging, monitoring For cancer diagnosis, staging, monitoring Evidence developed through clinical trials Evidence developed through clinical trials

32. 32 Coverage with Evidence Development NCDs PET for AD Sep 04 CRC NCI Trials Jan 05 ICDs PET 6 Cancers Jan 05 Cochlear Implant Apr 05 US Fracture Healing Apr 05

33. 33 Technical Assessments (TAs) Health tech assessment to meet policy or clinical objectives Health tech assessment to meet policy or clinical objectives Technology performance characteristics assessed Technology performance characteristics assessed Safety Safety Efficacy Efficacy Effectiveness Effectiveness Outcomes Outcomes Appropriateness Appropriateness Economic impacts Economic impacts

34. 34 Technical Assessments (TAs) Components Components Identifying & prioritizing technologies for assessment Identifying & prioritizing technologies for assessment Collecting & analyzing data Collecting & analyzing data Synthesizing & grading evidence Synthesizing & grading evidence Disseminating findings & recommendations Disseminating findings & recommendations Criteria for commissioning a TA Criteria for commissioning a TA Body of evidence extensive & timeframes in jeopardy Body of evidence extensive & timeframes in jeopardy Complexity of medical & scientific literature is great Complexity of medical & scientific literature is great Significant differences of opinion exist Significant differences of opinion exist Technical/clinical expertise or analytic methods great Technical/clinical expertise or analytic methods great

35. 35 TA Options Medicare Coverage Advisory Committee Medicare Coverage Advisory Committee External TA External TA AHRQ AHRQ Evidence-Based Practice Center (EPC) Evidence-Based Practice Center (EPC) Other qualified entity Other qualified entity For more information: For more information: www.cms.hhs.gov/coverage

36. 36 FDA Parallel Review 2005 HHS Medical Innovation Task Force report: “Moving Medical Innovations Forward—New Initiatives from HHS” 2005 HHS Medical Innovation Task Force report: “Moving Medical Innovations Forward—New Initiatives from HHS” Recommended increased collaboration between CMS and FDA in four areas Recommended increased collaboration between CMS and FDA in four areas Parallel review Parallel review Post-marketing surveillance Post-marketing surveillance Humanitarian Device Exemptions (HDEs) Humanitarian Device Exemptions (HDEs) Summaries of safety and effectiveness (SSEs) Summaries of safety and effectiveness (SSEs)

37. 37 FDA Parallel Review CMS & FDA currently discussing simultaneous evaluation of FDA-regulated medical products when the product sponsor and both CMS & FDA agree to such parallel review CMS & FDA currently discussing simultaneous evaluation of FDA-regulated medical products when the product sponsor and both CMS & FDA agree to such parallel review Federal Register notice expected soon for public comment Federal Register notice expected soon for public comment Pilot project being defined Pilot project being defined Several post-marketing surveillance projects in development Several post-marketing surveillance projects in development Provide FDA with access to claims data for monitoring safety or effectiveness of Part B devices, drugs, biologicals Provide FDA with access to claims data for monitoring safety or effectiveness of Part B devices, drugs, biologicals Part D expansion when data available Part D expansion when data available HDEs and SSEs not being discussed yet HDEs and SSEs not being discussed yet

38. 38 AHRQ EBM Activities Evidence-Based Practice Centers (EPCs) Evidence-Based Practice Centers (EPCs) 1997: AHRQ established 12 EPCs to promote EB practice in everyday care 1997: AHRQ established 12 EPCs to promote EB practice in everyday care Review relevant scientific literature on clinical, behavioral, and organization and financing topics to produce evidence reports & technology assessments Review relevant scientific literature on clinical, behavioral, and organization and financing topics to produce evidence reports & technology assessments Reports utilization: Reports utilization: Informing and developing coverage decisions Informing and developing coverage decisions Quality measures Quality measures Educational materials and tools Educational materials and tools Guidelines Guidelines Research agendas Research agendas

39. 39 AHRQ EBM Activities Effective Health Care Program Effective Health Care Program Medicare Modernization Act (MMA) Section 1013 Medicare Modernization Act (MMA) Section 1013 Authorizes AHRQ to support and conduct research with a focus on outcomes, comparative clinical effectiveness, and appropriateness of pharmaceuticals, devices and health care services Authorizes AHRQ to support and conduct research with a focus on outcomes, comparative clinical effectiveness, and appropriateness of pharmaceuticals, devices and health care services Research determined by needs of Medicare, Medicaid, and SCHIP programs Research determined by needs of Medicare, Medicaid, and SCHIP programs Approaches Approaches Synthesize knowledge: EPCs Synthesize knowledge: EPCs Generate knowledge: DEcIDE Research Network Generate knowledge: DEcIDE Research Network Translate knowledge: Clinical Decisions & Communications Science Center Translate knowledge: Clinical Decisions & Communications Science Center

40. 40 AHRQ Effective Health Care Topics Arthritis and non-traumatic joint disorders Arthritis and non-traumatic joint disorders Cancer Cancer Chronic obstructive pulmonary disease and asthma Chronic obstructive pulmonary disease and asthma Dementia including Alzheimer's disease Dementia including Alzheimer's disease Depression and other mood disorders Depression and other mood disorders Diabetes mellitus Diabetes mellitus Ischemic heart disease Ischemic heart disease Peptic ulcer disease and dyspepsia Peptic ulcer disease and dyspepsia Pneumonia Pneumonia Stroke and hypertension Stroke and hypertension

41. 41 AHRQ & NIH EBM Activities National Guideline Clearinghouse National Guideline Clearinghouse Partnership between AHRQ, AMA, AHIP Partnership between AHRQ, AMA, AHIP Guidelines compendium, comparison, syntheses Guidelines compendium, comparison, syntheses Partnering with CMS, NIH, FDA and other federal agencies to utilize EBM principles in quality and research activities Partnering with CMS, NIH, FDA and other federal agencies to utilize EBM principles in quality and research activities NIH NIH Translational and applied research Translational and applied research HRSA HRSA Organ Donation Breakthrough Initiative Organ Donation Breakthrough Initiative

42. 42 Contact Information Barry M. Straube, M.D. Acting Chief Medical Officer Acting Director, Office of Clinical Standards & Quality Centers for Medicare & Medicaid Services 7500 Security Boulevard Baltimore, MD 21244 Email: Barry.Straube@cms.hhs.gov Phone: (410) 786-6841