EvidenceBased Medicine Key Trends and Lessons Learned from CMS

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Evidence-Based Medicine Key Trends and Lessons
Learned from CMS

• Mass MEDIC
• Brian Carey
• April 20, 2006

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Overview

• Medicare coverage basics
• When is evidence adequate for coverage and
  reimbursement
• Coverage with evidence development
• Lessons learned from case studies

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CMS Quality Initiative

• Administrator Mark McClellan wants to transform
  CMS from a payer into a public health agency
• Promising new technologies may have insufficient
  evidence for informing clinical practice
• Premarket randomized clinical trials may be too
  limited to represent outcomes in real practice
• Health system seeks better outcomes at lowest
  possible costs
• Health IT will allow for great advances in data
  management, utility for research

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Statutory Basis for Coverage

• Sect. 1862 (a)(1)(A), Title 18, SSA
• Notwithstanding any other provisions of law . .
  .no payment may be madefor items or services . .
  which are not reasonable and necessary for the
  diagnosis or treatment of illness or injury.

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Reasonable and Necessary

• CMS has been developing evidence based framework
  for coverage decisions
• case law derived from NCDs since 2000
• Safe and effective FDA
• Adequate evidence to conclude that the item or
  service improves net health outcomes
• generalizable to the Medicare population
• as good or better than current covered
  alternatives

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CMS Follows General Principles of EBM

• Published and non-published studies, expert
  opinion, technology assessments, professional
  societies, recommendations from the Medicare
  Coverage Advisory Committee (MCAC)
• Key areas of focus include
• Methodological considerations, including study
  design, implementation, and analysis
• Relevance of chosen outcomes preference for
  those experienced by the patient
• Generalizable to the Medicare population
• Qualitative assessment of net risks and benefits
  based on individual studies

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Ladder of Evidential Strength

• ________Meta-analysis of individual patient data
• ___________ Large, multi-center RCTs
• ______________ Meta-analysis of grouped
  data
• ______________ Smaller, single site
  RCTs
• ______________ Prospective
  cohort studies
• ____________ retrospective cohort
• ____________ Poorly controlled
• studies
  _______________Uncontrolled
• studies (case-series or
• reports)

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CMS Does Not Consider Cost Effectiveness

• CMS released Guidance document on April 11, 2006
  on NCD Process
• Cost effectiveness is not a factor CMS considers
  in making NCDs. In other words, the cost of a
  particular technology is not relevant in the
  determination of whether the technology improves
  health outcomes or should be covered for the
  Medicare population through an NCD.

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Frequently Unanswered Questions

• Adverse events in broad use
• Off-label uses or new combinations of approved
  uses
• Risks and benefits in subgroups
• Outcomes for patient and provider types excluded
  from trials
• Comparative effectiveness
• Outcomes not measured in trials
• Clinical utility of diagnostic tests
• Surgery, some devices and other technologies with
  limited regulatory review

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Practical Clinical Trials

• CMS encourages use of data from Practical
  Clinical Trials (JAMA article by Dr. Tunis)
• Greater emphasis on health outcomes actually
  experienced by patients, such as quality of life,
  functional status, duration of disability,
  morbidity and mortality
• less emphasis on outcomes that patients do not
  directly experience, such as changes in
  laboratory values, radiographic response,
  sensitivity/specificity, physiologic parameters
  and other intermediate or surrogate outcomes

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Case Study Coverage of MTWA

• Microvolt T-Wave Alternans is a non-diagnostic
  test for stratification of patients who may be at
  risk of sudden cardiac death
• MTWA had limited Medicare coverage that varied by
  local carrier
• Cambridge Heart requested NCD in 2005 and met
  with CMS numerous times

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MTWA Coverage

• CMS reviewed peer reviewed article and existing
  technology assessment
• Out of 1028 citations in support of MTWA CMS
  focused on 12 peer reviewed articles
• CMS performed systematic review of literature
• Final NCD issued in March, 2006 expanding
  coverage of MTWA using spectral analytic method.

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Observations

• BCBS TA had concluded that MTWA did not meet
  criteria for coverage. CMS focused on Medicare
  eligible population
• CMS found there was not sufficient evidence to
  cover modified moving average (MMA) technique for
  determining MTWA
• Aetna followed CMSs policy

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CMS Coverage with Evidence Development (CED)

• Support creation of better evidence
• Links Medicare coverage with requirement for
  prospective data collection
• Builds on existing Evidence Based Medicine
  coverage framework

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CMS Evidence Objectives

• Provide more and better information for health
  care decision makers
• Expand capacity of clinical research enterprise
  to produce information oriented to decision
  makers
• Support innovation while addressing frequently
  unanswered questions
• Move toward system in which care delivery and
  evaluation are more integrated

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Use of CED by CMS

• Lung volume reduction surgery
• PET for suspected dementia
• ICD for primary prevention of SCD
• PET for non-covered oncology uses

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Case Study National Oncologic PET Registry

• What is a CMS approved Coverage with Evidence
  Development Program
• In 2000, the Centers for Medicare and Medicaid
  Services (CMS) expanded its coverage of positron
  emission tomography (PET) with F-18
  fluorodeoxyglucose (FDG) to a wide variety of
  indications for several common cancers but not
  all cancers.
• In November 2004, CMS proposed expanding PET
  coverage to most other cancers, if providers
  collect relevant data in a CMS-approved clinical
  registry.

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National Oncologic PET Registry (Cont.)

• NOPR is rune by American College of Radiology and
  Academy of Molecular Imaging
• All PET facilities can participate (for a fee)
• Requires Pre-PET and Post-PET
• Case report forms completed timely
• Will assess change in intended management

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Lessons Learned

• CED policy can result in significant expansion
• Registries are very complicated numerous
  logistic issues such as IRB, informed consent and
  Privacy Act
• CMS likely to issue new guidance on CED

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Section 1013 Clinical Effectiveness Studies

• MMA included specific provision on the
  comparative clinical effectiveness and
  appropriateness of health care items
• HHS developed 10 priority areas
• Agency for Healthcare Research and Quality (AHRQ)
  is conducting studies through EPCs
• Systematic reviews and syntheses of the
  scientific literature

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Section 1013 AHRQ Studies

• Management strategies for gastroesophageal reflux
  disease
• Benefits and safety of analgesics for
  osteoarthritis
• New diagnostic technologies for evaluation of
  abnormal breast cancer screening
• Epoetin and Darbepoetin for managing anemia in
  patients undergoing cancer treatment
• Off-label use of atypical anti-psychotic
  medications
• Renal artery stenting compared to aggressive
  anti-hypertensive medical therapy for mild renal
  artery stenosis
• Therapies for localized prostate cancer
• Oral medications for diabetes management
• Medications for depression management
• Drug therapies and behavioral interventions for
  osteoporosis and osteopenia

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EBM at State Level

• States have been further ahead of CMS
• Center for Evidence-based Policy at Oregon Health
  and Science University
• Performed systematic reviews for 10 to 15 states
  on drug effectiveness
• Starting to look at devices such as imaging and
  diagnostic tests

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Conclusion Recent Trends

• Increased transparency of coverage process
• Focus on evidence and data generation beyond what
  has traditionally been required for FDA approval
• Initiative to increase data collection to use for
  future coverage decisions or refinements
• Increased collaboration between CMS and other
  government agencies such as FDA and NCI

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Contact info

• Brian P. Carey
• Foley Hoag LLP
• bcarey_at_foleyhoag.com
• [phone]
• [phone]