Download by CMS - MassMEDIC

Evidence-Based Medicine – Key Trends and
Lessons Learned from CMS
Mass MEDIC
Brian Carey
April 20, 2006
Overview
 Medicare coverage basics
 When is evidence adequate for coverage and
reimbursement
 Coverage with evidence development
 Lessons learned from case studies
CMS Quality Initiative
 Administrator Mark McClellan wants to transform CMS
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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
Statutory Basis for Coverage
 Sect. 1862 (a)(1)(A), Title 18, SSA
 “Notwithstanding any other provisions of law
. . .no payment may be made…for items or
services . . [which] are not reasonable and
necessary for the diagnosis or treatment of
illness or injury.”
Reasonable and Necessary
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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
CMS Follows General Principles of
EBM
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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
Ladder of Evidential Strength
Higher
STRENGTH OF
EVIDENCE
Lower
________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)
CMS Does Not Consider Cost
Effectiveness
 CMS released Guidance document on April 11,
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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.”
Frequently Unanswered Questions
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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
Practical Clinical Trials
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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
Case Study – Coverage of MTWA
 Microvolt T-Wave Alternans is a nondiagnostic 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
MTWA Coverage
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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.
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 CMS’s policy
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
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
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
Case Study – National Oncologic PET
Registry
 What is a CMS approved “Coverage with Evidence
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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.
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
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
Section 1013 – Clinical Effectiveness
Studies
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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
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
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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
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
Conclusion – Recent Trends
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Increased transparency of coverage process
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Initiative to increase data collection to use for future
coverage decisions or refinements
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Increased collaboration between CMS and other
government agencies such as FDA and NCI
Focus on evidence and data generation beyond what
has traditionally been required for FDA approval
Contact info
Brian P. Carey
Foley Hoag LLP
[email protected]
(617) 832-1712
(202) 261-7398