The Latest Federal Initiatives in Evidence-Based Medicine

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

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

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

7. A Variation Problem Dartmouth Atlas of Healthcare

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

11. CMS Quality Roadmap: Strategies • Work through partnerships to achieve specific quality goals • Publish quality measurements and information as a basis for supporting more effective quality improvement efforts • 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. CMS Quality Roadmap: Strategies for QI • Assist practitioners in making care more effective and less costly, especially by promoting the adoption of HIT • 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. Centers for Medicare & Medicaid Services (CMS) CMS as a Public Health Agency • Using CMS influence and financial leverage to transform American healthcare system • Quality, Value, Efficiency, Cost-effectiveness • Assisting patients and providers in receiving evidence-based, technologically-advanced care

14. 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.” D.L. Sackett et al: BMJ 312, no.7023 (1996):71-72

15. 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.” D.L. Sacket et al, BMJ 312, no.7023 (1996): 71-72

16. Evidence-Based Medicine • Evidence-Based Guidelines (EBGs) • Different methods for designing guidelines • Global subjective judgment • Consensus-based • Evidence-based • Outcomes-based • Preference-based • 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

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 • Utilization of an explicit, rigorous process • End “product” is generic, applying to a class or group of patients, not individual patients • 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

18. Evidence-Based Medicine • Evidence-Based Individual Decision Making (EBID) • Focus on educating clinicians to help them bring more research and evidence into individual decisions about individual patients • Integrating EBGs with EBID • 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.

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

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

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

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

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 • Juxtaposed to FDA “safe and effective” standards • Binding on all contractors (supersedes any local coverage policies)

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

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

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

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

28. Coverage with Evidence Development (CED) • 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 • Offers prompt coverage linked with more evidence development • Speeds access, safeguards patients, improves evidence for better decisions

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

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

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

32. Coverage with Evidence Development NCDs

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

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

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

36. FDA Parallel Review • 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 • Parallel review • Post-marketing surveillance • Humanitarian Device Exemptions (HDEs) • Summaries of safety and effectiveness (SSEs)

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 • Federal Register notice expected soon for public comment • Pilot project being defined • 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 • Part D expansion when data available • HDEs and SSEs not being discussed yet

38. AHRQ EBM Activities • Evidence-Based Practice Centers (EPCs) • 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 • Reports utilization: • Informing and developing coverage decisions • Quality measures • Educational materials and tools • Guidelines • Research agendas

39. AHRQ EBM Activities • Effective Health Care Program • 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 • Research determined by needs of Medicare, Medicaid, and SCHIP programs • Approaches • Synthesize knowledge: EPCs • Generate knowledge: DEcIDE Research Network • Translate knowledge: Clinical Decisions & Communications Science Center

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

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

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