The Latest Federal The Latest Federal Initiatives in Evidence-Initiatives in Evidence-

Based MedicineBased Medicine

Barry M. Straube, M.D.Barry M. Straube, M.D.

Centers for Medicare & Medicaid Centers for Medicare & Medicaid ServicesServices

Citizen’s Council on Health CareCitizen’s Council on Health Care

November 10, 2005November 10, 2005

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Centers for Medicare & Centers for Medicare & Medicaid Services (CMS)Medicaid Services (CMS)

Provides health benefits for over Provides health benefits for over 76 million Americans76 million Americans MedicareMedicare MedicaidMedicaid State Children's Health Insurance State Children's Health Insurance

Program (SCHIP)Program (SCHIP) Spends over $600 billion annually Spends over $600 billion annually

for health care services in CMS for health care services in CMS programsprograms

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Centers for Medicare & Centers for Medicare & Medicaid Services (CMS)Medicaid Services (CMS)

Healthcare Benefits AdministrationHealthcare Benefits Administration Establish payment methodology for providersEstablish payment methodology for providers Conduct research on financing, treatment & Conduct research on financing, treatment &

managementmanagement Assure that contractors & state agencies run CMS Assure that contractors & state agencies run CMS

programs correctlyprograms correctly Identify fraud & abuse, take appropriate actionIdentify fraud & abuse, take appropriate action

Beneficiary Focused ActivitiesBeneficiary Focused Activities Benefits and health educationBenefits and health education Healthcare data for choiceHealthcare data for choice Advocacy: Appeals, Grievances, patient rightsAdvocacy: Appeals, Grievances, patient rights Preventive servicesPreventive services

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Centers for Medicare & Centers for Medicare & Medicaid Services (CMS)Medicaid Services (CMS)

Quality-Focused ActivitiesQuality-Focused Activities Laboratory Testing (CLIA)Laboratory Testing (CLIA) Survey & Certification of Health Care Facilities Survey & Certification of Health Care Facilities

(LTC, SNF, HHA, Hospitals)(LTC, SNF, HHA, Hospitals) End-Stage Renal Disease NetworksEnd-Stage Renal Disease Networks Organ Procurement OrganizationsOrgan Procurement Organizations Quality Improvement OrganizationsQuality Improvement Organizations Multiple Demonstration ProjectsMultiple Demonstration Projects Health Information Technology promotion/adoptionHealth Information Technology promotion/adoption Pay-for-Performance, Paying for Quality, Value-Pay-for-Performance, Paying for Quality, Value-

Based PurchasingBased Purchasing Healthcare data, public reportingHealthcare data, public reporting Evidence-Based Medicine, Technology & InnovationEvidence-Based Medicine, Technology & Innovation

A Variation ProblemA Variation Problem

Dartmouth Atlas of Healthcare

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CMS Quality RoadmapCMS Quality Roadmap VISION: VISION: The right care for The right care for

every person every timeevery person every time Make care:Make care:

Safe Safe Effective Effective Efficient Efficient Patient-centered Patient-centered TimelyTimelyEquitableEquitable

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CMS Quality Roadmap: CMS Quality Roadmap: StrategiesStrategies

1.1. Work through partnerships to achieve Work through partnerships to achieve specific quality goalsspecific quality goals

2.2. Publish quality measurements and Publish quality measurements and information as a basis for supporting information as a basis for supporting more effective quality improvement more effective quality improvement effortsefforts

3.3. Pay in a way that expresses our Pay in a way that expresses our commitment to quality, and that helps commitment to quality, and that helps providers and patients to take steps to providers and patients to take steps to improve health and avoid unnecessary improve health and avoid unnecessary costscosts

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CMS Quality Roadmap: CMS Quality Roadmap: Strategies for QIStrategies for QI

4.4. Assist practitioners in making care Assist practitioners in making care more effective and less costly, more effective and less costly, especially by promoting the adoption especially by promoting the adoption of HITof HIT

5.5. Bring effective new treatments to Bring effective new treatments to patients more rapidly and help develop patients more rapidly and help develop better evidence so that doctors and better evidence so that doctors and patients can use medical technologies patients can use medical technologies and treatments more effectivelyand treatments more effectively

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Centers for Medicare & Centers for Medicare & Medicaid Services (CMS)Medicaid Services (CMS)

CMS as a Public Health AgencyCMS as a Public Health Agency Using CMS influence and financial Using CMS influence and financial

leverage to transform American healthcare leverage to transform American healthcare systemsystem

Quality, Value, Efficiency, Cost-Quality, Value, Efficiency, Cost-effectivenesseffectiveness

Assisting patients and providers in Assisting patients and providers in receiving evidence-based, technologically-receiving evidence-based, technologically-advanced careadvanced care

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

Evidence-Based MedicineEvidence-Based Medicine

““Evidence-based medicineEvidence-based medicine is is the conscientious, explicit, the conscientious, explicit,

and judicious use of current and judicious use of current best evidence in making best evidence in making

decisions about the care of decisions about the care of individual patients.”individual patients.”

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

Evidence-Based MedicineEvidence-Based Medicine

““The practice of evidence-based The practice of evidence-based medicine means integrating medicine means integrating individual clinical expertise individual clinical expertise

with the best available external with the best available external clinical evidence from clinical evidence from

systematic research and our systematic research and our patient’s unique value and patient’s unique value and

circumstances.”circumstances.”

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Evidence-Based MedicineEvidence-Based Medicine Evidence-Based Guidelines (EBGs)Evidence-Based Guidelines (EBGs)

Different methods for designing guidelinesDifferent methods for designing guidelines Global subjective judgmentGlobal subjective judgment Consensus-basedConsensus-based Evidence-basedEvidence-based Outcomes-basedOutcomes-based Preference-basedPreference-based

Guidelines should be based on evidence, not Guidelines should be based on evidence, not subjective judgment or opinionsubjective judgment or opinion

““Evidence-based”: coverage, performance Evidence-based”: coverage, performance measures, medical necessity, benefit design, measures, medical necessity, benefit design, disease management, quality improvement, disease management, quality improvement, regulations, & public policiesregulations, & public policies

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Features of EBGsFeatures of EBGs Work of analyzing evidence and Work of analyzing evidence and

developing a guideline or policy is done by developing a guideline or policy is done by a small group of experts, usually a small group of experts, usually sponsored by an organizationsponsored by an organization

Utilization of an explicit, rigorous processUtilization of an explicit, rigorous process End “product” is generic, applying to a End “product” is generic, applying to a

class or group of patients, not individual class or group of patients, not individual patientspatients

Effects on care are indirectEffects on care are indirect Enable, motivate or (sometimes) force Enable, motivate or (sometimes) force

providers to deliver certain types of care to providers to deliver certain types of care to groups of people, NOT to a particular patientgroups of people, NOT to a particular patient

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Evidence-Based MedicineEvidence-Based Medicine Evidence-Based Individual Decision Making Evidence-Based Individual Decision Making

(EBID)(EBID) Focus on educating clinicians to help them Focus on educating clinicians to help them

bring more research and evidence into bring more research and evidence into individual decisions about individual patientsindividual decisions about individual patients

Integrating EBGs with EBIDIntegrating EBGs with EBID EBM should not just focus on individual EBM should not just focus on individual

physicians and their decisionsphysicians and their decisions EBM is a set of principles and methods intended EBM is a set of principles and methods intended

to ensure that to the greatest extent possible, to ensure that to the greatest extent possible, medical decisions, guidelines, and other types of medical decisions, guidelines, and other types of policies are based on and consistent with good policies are based on and consistent with good evidence of effectiveness and benefit.evidence of effectiveness and benefit.

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CMS Evidence-Based CMS Evidence-Based InitiativesInitiatives

National Coverage DecisionsNational Coverage Decisions Coverage with Evidence DevelopmentCoverage with Evidence Development

Medicare Benefit Categories and specific Medicare Benefit Categories and specific benefitsbenefits Preventive Care ServicesPreventive Care Services

Technology & Innovation AssessmentsTechnology & Innovation Assessments Medical DevicesMedical Devices TherapeuticsTherapeutics DiagnosticsDiagnostics

Health Information TechnologyHealth Information Technology Data collection via EHRs, registries, eprescribing, etc.Data collection via EHRs, registries, eprescribing, etc. Clinical Decision SupportClinical Decision Support

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CMS Evidence-Based CMS Evidence-Based InitiativesInitiatives

Quality & Efficiency Measure developmentQuality & Efficiency Measure development Quality AlliancesQuality Alliances National Quality ForumNational Quality Forum Constant reassessmentConstant reassessment

Drug Formulary for Medicare Prescription Drug Formulary for Medicare Prescription Drug BenefitDrug Benefit

Pay-for-Performance InitiativesPay-for-Performance Initiatives HospitalHospital PhysicianPhysician Home Health, Skilled Nursing Facilities, Home Health, Skilled Nursing Facilities,

ESRD facilitiesESRD facilities

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CMS EBM InitiativesCMS EBM Initiatives

QIO and ESRD Network Quality QIO and ESRD Network Quality Improvement ActivitiesImprovement Activities

CMS Breakthrough InitiativesCMS Breakthrough Initiatives Fistula FirstFistula First Surgical Care Improvement Program Surgical Care Improvement Program

(SCIP)(SCIP) Nursing Home InitiativesNursing Home Initiatives

Restraint reductionRestraint reduction Pressure ulcers reductionPressure ulcers reduction Pain controlPain control Staff turnover rate reductionStaff turnover rate reduction

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Steps to Coverage Steps to Coverage Determination and Determination and

Payment Payment Outside of CMSOutside of CMS::

Congress determines benefit categories Congress determines benefit categories FDA approves drugs/devices for marketFDA approves drugs/devices for market

Within CMSWithin CMS:: Benefit Category Determination Benefit Category Determination Coverage DecisionCoverage Decision Coding AssignmentCoding Assignment Payment DeterminationPayment Determination

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National Coverage National Coverage Determinations (NCDs)Determinations (NCDs)

Evidence-based decisions on whether to Evidence-based decisions on whether to add or revise coverage for an item or add or revise coverage for an item or service under Medicare Part A or Bservice under Medicare Part A or B

Must meet statutory “reasonable and Must meet statutory “reasonable and necessary” standardnecessary” standard Juxtaposed to FDA “safe and effective” standardsJuxtaposed to FDA “safe and effective” standards

Binding on all contractors (supersedes any Binding on all contractors (supersedes any local coverage policies)local coverage policies)

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Most Coverage is Most Coverage is LocalLocal

Local90%

National10%

Local

National

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What prompts NCDs?What prompts NCDs?

Internal (20%) or external (80%) Internal (20%) or external (80%) requestsrequests

May involve:May involve: New technology or treatment not New technology or treatment not

addressed in pastaddressed in past Reconsideration of a prior non-Reconsideration of a prior non-

coverage (or limited coverage) coverage (or limited coverage) decisiondecision

Response to significant variation in Response to significant variation in local coverage policieslocal coverage policies

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Key Factors Considered in Key Factors Considered in National Coverage National Coverage Determinations Determinations

• Must be potentially a benefit of Must be potentially a benefit of MedicareMedicare

• Evidence of improved health outcomes Evidence of improved health outcomes • Appropriate for Medicare populationAppropriate for Medicare population• Replicable in provider communityReplicable in provider community• Costs not a primary factor historicallyCosts not a primary factor historically

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Recent NCD Guidance Documents

CMS requested public comment on CMS requested public comment on factors to consider infactors to consider in::

Evidence development methodsEvidence development methods Process for study design and Process for study design and

implementationimplementation RegistriesRegistries Clinical trialsClinical trials Other methodsOther methods

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Coverage with Evidence Coverage with Evidence Development (CED) Development (CED)

Used for promising innovations with Used for promising innovations with insufficient evidence for individual insufficient evidence for individual patients or the Medicare populationpatients or the Medicare population

Also used when conclusive evidence is Also used when conclusive evidence is not available, but existing evidence not available, but existing evidence strongly suggests probable benefitstrongly suggests probable benefit

Offers prompt coverage linked with Offers prompt coverage linked with more evidence developmentmore evidence development

Speeds access, safeguards patients, Speeds access, safeguards patients, improves evidence for better decisionsimproves evidence for better decisions

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Coverage With Evidence Coverage With Evidence Development = Development = an alternative an alternative

to non-coverageto non-coverage

Is existing evidence sufficient to support Medicare coverage?

Coverage

Yes

No

Coverage with Evidence Development

Non-coverage

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Flexible Coverage ProcessesFlexible Coverage ProcessesExamplesExamples

Prophylactic implantable Prophylactic implantable cardioverter defibrillator (ICD) cardioverter defibrillator (ICD) Data submitted to national registries, Data submitted to national registries,

at low cost for participating hospitals at low cost for participating hospitals all over the countryall over the country

Otherwise would have more limited Otherwise would have more limited coveragecoverage

Expanded coverage to reach more Expanded coverage to reach more patientspatients

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Flexible Coverage ProcessesFlexible Coverage ProcessesExamplesExamples

Additional off-label uses of cancer drugsAdditional off-label uses of cancer drugs No FDA-approved results, and no studies No FDA-approved results, and no studies

covered in medical referencescovered in medical references Previously would not have been nationally Previously would not have been nationally

coveredcovered Evidence developed through clinical trialsEvidence developed through clinical trials

FDG-PET scanning FDG-PET scanning For dementia and neurodegenerative For dementia and neurodegenerative

disordersdisorders For cancer diagnosis, staging, monitoringFor cancer diagnosis, staging, monitoring Evidence developed through clinical trialsEvidence developed through clinical trials

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Coverage with Evidence Coverage with Evidence Development NCDsDevelopment NCDs

PET for ADPET for AD Sep 04Sep 04

CRC NCI TrialsCRC NCI Trials Jan 05Jan 05

ICDsICDs Jan 05Jan 05

PET 6 CancersPET 6 Cancers Jan 05Jan 05

Cochlear ImplantCochlear Implant Apr 05Apr 05

US Fracture HealingUS Fracture Healing Apr 05Apr 05

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Technical Assessments Technical Assessments (TAs)(TAs)

Health tech assessment to meet Health tech assessment to meet policy or clinical objectivespolicy or clinical objectives

Technology performance Technology performance characteristics assessedcharacteristics assessed SafetySafety EfficacyEfficacy EffectivenessEffectiveness OutcomesOutcomes AppropriatenessAppropriateness Economic impactsEconomic impacts

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Technical Assessments Technical Assessments (TAs)(TAs)

ComponentsComponents Identifying & prioritizing technologies for Identifying & prioritizing technologies for

assessmentassessment Collecting & analyzing dataCollecting & analyzing data Synthesizing & grading evidenceSynthesizing & grading evidence Disseminating findings & recommendationsDisseminating findings & recommendations

Criteria for commissioning a TACriteria for commissioning a TA Body of evidence extensive & timeframes in Body of evidence extensive & timeframes in

jeopardyjeopardy Complexity of medical & scientific literature is Complexity of medical & scientific literature is

greatgreat Significant differences of opinion existSignificant differences of opinion exist Technical/clinical expertise or analytic methods Technical/clinical expertise or analytic methods

greatgreat

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TA OptionsTA Options

Medicare Coverage Advisory Medicare Coverage Advisory CommitteeCommittee

External TAExternal TA AHRQAHRQ Evidence-Based Practice Center (EPC)Evidence-Based Practice Center (EPC) Other qualified entityOther qualified entity

For more information:For more information:

www.cms.hhs.gov/coveragewww.cms.hhs.gov/coverage

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FDA Parallel ReviewFDA Parallel Review 2005 HHS Medical Innovation Task Force 2005 HHS Medical Innovation Task Force

report: “Moving Medical Innovations report: “Moving Medical Innovations Forward—New Initiatives from HHS”Forward—New Initiatives from HHS”

Recommended increased collaboration Recommended increased collaboration between CMS and FDA in four areasbetween CMS and FDA in four areas Parallel reviewParallel review Post-marketing surveillancePost-marketing surveillance Humanitarian Device Exemptions (HDEs)Humanitarian Device Exemptions (HDEs) Summaries of safety and effectiveness (SSEs)Summaries of safety and effectiveness (SSEs)

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FDA Parallel ReviewFDA Parallel Review CMS & FDA currently discussing simultaneous CMS & FDA currently discussing simultaneous

evaluation of FDA-regulated medical products evaluation of FDA-regulated medical products when the product sponsor and both CMS & when the product sponsor and both CMS & FDA agree to such parallel reviewFDA agree to such parallel review Federal Register notice expected soon for public Federal Register notice expected soon for public

commentcomment Pilot project being definedPilot project being defined

Several post-marketing surveillance projects in Several post-marketing surveillance projects in developmentdevelopment Provide FDA with access to claims data for Provide FDA with access to claims data for

monitoring safety or effectiveness of Part B monitoring safety or effectiveness of Part B devices, drugs, biologicalsdevices, drugs, biologicals

Part D expansion when data availablePart D expansion when data available HDEs and SSEs not being discussed yetHDEs and SSEs not being discussed yet

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AHRQ EBM ActivitiesAHRQ EBM Activities Evidence-Based Practice Centers (EPCs)Evidence-Based Practice Centers (EPCs)

1997: AHRQ established 12 EPCs to promote EB 1997: AHRQ established 12 EPCs to promote EB practice in everyday carepractice in everyday care

Review relevant scientific literature on clinical, Review relevant scientific literature on clinical, behavioral, and organization and financing topics behavioral, and organization and financing topics to produce evidence reports & technology to produce evidence reports & technology assessmentsassessments

Reports utilization:Reports utilization: Informing and developing coverage decisionsInforming and developing coverage decisions Quality measuresQuality measures Educational materials and toolsEducational materials and tools GuidelinesGuidelines Research agendasResearch agendas

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AHRQ EBM ActivitiesAHRQ EBM Activities Effective Health Care ProgramEffective Health Care Program

Medicare Modernization Act (MMA) Section 1013Medicare Modernization Act (MMA) Section 1013 Authorizes AHRQ to support and conduct research Authorizes AHRQ to support and conduct research

with a focus on outcomes, comparative clinical with a focus on outcomes, comparative clinical effectiveness, and appropriateness of effectiveness, and appropriateness of pharmaceuticals, devices and health care servicespharmaceuticals, devices and health care services

Research determined by needs of Medicare, Research determined by needs of Medicare, Medicaid, and SCHIP programsMedicaid, and SCHIP programs

ApproachesApproaches Synthesize knowledge: EPCsSynthesize knowledge: EPCs Generate knowledge: DEcIDE Research NetworkGenerate knowledge: DEcIDE Research Network Translate knowledge: Clinical Decisions & Translate knowledge: Clinical Decisions &

Communications Science CenterCommunications Science Center

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AHRQ Effective Health AHRQ Effective Health Care TopicsCare 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 hypertensionStroke and hypertension

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AHRQ & NIH EBM AHRQ & NIH EBM ActivitiesActivities

National Guideline ClearinghouseNational Guideline Clearinghouse Partnership between AHRQ, AMA, AHIPPartnership between AHRQ, AMA, AHIP Guidelines compendium, comparison, synthesesGuidelines compendium, comparison, syntheses

Partnering with CMS, NIH, FDA and other Partnering with CMS, NIH, FDA and other federal agencies to utilize EBM principles federal agencies to utilize EBM principles in quality and research activitiesin quality and research activities

NIHNIH Translational and applied researchTranslational and applied research

HRSAHRSA Organ Donation Breakthrough InitiativeOrgan Donation Breakthrough Initiative

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Contact InformationContact Information

Barry M. Straube, M.D.Barry M. Straube, M.D.

Acting Chief Medical OfficerActing Chief Medical Officer

Acting Director, Office of Clinical Standards Acting Director, Office of Clinical Standards & Quality& Quality

Centers for Medicare & Medicaid ServicesCenters for Medicare & Medicaid Services

7500 Security Boulevard7500 Security Boulevard

Baltimore, MD 21244Baltimore, MD 21244

Email: Barry.Straube@cms.hhs.govEmail: Barry.Straube@cms.hhs.gov

Phone: (410) 786-6841Phone: (410) 786-6841