National Health Policy Conference
Using Evidence to Design Benefits
February 9, 2010
Washington, DC
Clifford Goodman, Ph.D.
Vice President
The Lewin Group
Falls Church, Virginia USA 22042
clifford.goodman@lewin.com
The Lewin Group
DO NOT TRANSMIT OR REPRODUCE WITHOUT PERMISSION
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Medicare Evidence Development & Coverage
Advisory Committee (MEDCAC)
• Provides independent guidance and expert advice to
CMS on specific clinical topics
• Supplements CMS' internal expertise; allows
unbiased, current deliberation of "state of the art"
technology and science
• Reviews and evaluates evidence and related
information on effectiveness and appropriateness of
medical items and services that are covered, or may
be eligible for coverage, under Medicare
• Judges strength of the available evidence and makes
recommendations to CMS based on that evidence
The Lewin Group
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Medicare National Coverage Process
Preliminary
Discussions
Reconsideration
Benefit
Category
National
Coverage
Request
6 months
30 days
Draft Decision
Memorandum
Posted
Staff
Review
External
Technology
Assessment
MEDCAC
Staff
Review
60 days
Public
Comments
Final Decision
Memorandum &
Implementation
Instructions
Department
Appeals
Board
9 months
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June 2009
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MEDCAC: Catheter Ablation for the Treatment of
Atrial Fibrillation, Oct. 21, 2009
1. How confident are you that the evidence is adequate to draw
conclusions about the health outcomes of interest to patients
treated with catheter ablation for atrial fibrillation?
– Recurrence of arrhythmia
– Symptom relief
– Stroke
– Survival
2. How confident are you that catheter ablation for the treatment of
atrial fibrillation improves health outcomes compared to other
therapies or treatments in the following populations:
– As first-line therapy?
– As second-line therapy?
– For first detected atrial fibrillation?
– For long-standing (greater than 1 year) atrial fibrillation?
– For paroxysmal atrial fibrillation?
– For persistent atrial fibrillation?
The Lewin Group
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MEDCAC: Catheter Ablation for the Treatment of
Atrial Fibrillation, Oct. 21, 2009
3. How confident are you that ablation improves long-term (greater
than 1 year) health outcomes?
4. How confident are you that the outcomes can be extrapolated to:
– Patients outside a controlled clinical study?
– The Medicare beneficiary population (age 65 years and older,
56% female)?
5. How confident are you that additional evidence is needed?
Discussion - Additional evidence, if needed:
• What type of additional evidence is needed to determine health
outcomes?
• What study designs are most appropriate to obtain this additional
evidence?
The Lewin Group
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MEDCAC: Pharmacogenomic Testing for Anticancer
Therapies, Jan. 27, 2010
1. How confident are you that there is sufficient evidence to
determine whether pharmacogenomic testing affects health
outcomes (including benefits and harms) for patients with cancer
whose anticancer treatment strategy is guided by the results of
testing as described below? (Please consider this question
separately for each test in the following clinical situations.)
a) CYP2D6 for breast cancer patients who are candidates for
tamoxifen
b) UGT1A1 for colon cancer patients who are candidates for
irinotecan
c) HER2/neu for breast cancer patients who are candidates for
trastuzumab
d) BCR-ABL for chronic myelogenous leukemia patients who are
candidates for imatinib
e) K-ras for metastatic colorectal cancer patients who are
candidates for cetuximab and/or panitumumab
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MEDCAC: Pharmacogenomic Testing for Anticancer
Therapies, Jan. 27, 2010
2. For those items where the answer to Question 1 is at least in the
Intermediate range (mean score > 2.5), how confident are you that
pharmacogenomic testing improves health outcomes for patients
with cancer whose anticancer treatment strategy is guided by the
results of testing as described below? (Please consider this
question separately for each test in the following clinical
situations.)
3. How confident are you that these conclusions are generalizable to
a. community based settings;
b. the Medicare beneficiary population?
4. Please discuss any important evidence gaps and recommend how
they should be addressed.
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Analytic Framework: Diagnostic Test and Alternative
Treatments
7
6
Symptomatic
Population
Dx Test
Detection
of Target
Condition
1
Alternative
Treatments
A
B
3
Intermediate
Outcomes

5


Mortality
Morbidity
Quality of Life
2
4
Adverse Effects
1.
2.
3.
4.
5.
6.
7.
Adverse Effects
of A or B
Is diagnostic test accurate for target condition?
Does diagnostic test result in adverse effects?
Do treatments change intermediate outcomes?
Do treatments result in adverse effects?
Are changes in intermediate outcomes associated with changes in health outcomes?
Does treatment improve health outcomes?
Is there direct evidence that diagnostic test improves health outcomes?
The Lewin Group
Adapted from: Harris, Helfand, Woolf, et al. 2001
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Analytical Framework: CYP450 for SSRIs
Source: Teutsch SM et al. EGAPP Working Group. Genet Med 2009;11(1):314.
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Hierarchies for Evaluating Genetic Tests and Other
Genomic Applications - EGAPP
Source: Teutsch SM et al. EGAPP Working Group. Genet Med 2009;11(1):3-14.
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Hierarchies for Evaluating Genetic Tests and Other
Genomic Applications - EGAPP
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Source: Teutsch SM et al. EGAPP Working Group. Genet Med 2009;11(1):314.
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CMS Findings on Genetic Testing for Warfarin
Anticoagulation Response
• In 2007, the FDA determined that available PGx
information warranted a change in the labeling of
warfarin to call attention to the potential relevance
of genetic information to prescribing of warfarin.1
• Regarding Medicare coverage, CMS stated in 2009
that …
1FDA
approves updated warfarin (Coumadin) prescribing information. Press release
of the Food and Drug Administration, Rockville, MD, August 16, 2007.
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CMS Findings on Genetic Testing for Warfarin
Anticoagulation Response
•
“CMS found no evidence that genetic testing can replace
PT/INR [prothrombin time/International Normalized Ratio
Testing] for titrating and monitoring warfarin therapy…. [W]e
propose that the evidence is insufficient to determine that
pharmacogenomic testing to predict warfarin responsiveness
improves patient oriented health outcomes related to the
underlying indication for warfarin anticoagulation or adverse
events related to warfarin therapy itself. In addition, we
propose that the evidence is insufficient to determine that
pharmacogenomic testing to predict warfarin responsiveness
leads to changes in physician management of beneficiaries’
anticoagulation therapy that would result in positive
outcomes.”2
2CMS.
Proposed Decision Memo for Pharmacogenomic Testing for Warfarin
Response (CAG-00400N) May 4, 2009.
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CMS Findings on Genetic Testing for Warfarin
Anticoagulation Response
•
•
Noting that the testing is promising, CMS proposed
“coverage with evidence development” in which Medicare
would cover the test only for beneficiaries enrolled in an RCT
meeting certain specifications, one of which is “The research
study protocol must explicitly discuss subpopulations
affected by the treatment under investigation.” Such RCTs
would provide further evidence that could inform a revised
coverage determination.
Other research continues on how genotypes affect sensitivity
to warfarin and how well genetic tests predict safer and more
effective doses of warfarin, including a large, multicenter RCT
designed to determine whether genetic information provides
additional benefit to what can be accomplished with
traditional clinically-based warfarin information alone.1
1Shurin
SB, Nabel EG. Pharmacogenomics--ready for prime time? N Engl J Med
2008;358(10):1061-3.
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Looking to Evidence Experts - US
AHRQ
• Effective Health Care Program
• Evidence-Based Practice Centers Program
• Technology Assessment Program
• DEcIDE Network
• USPSTF
CDC
• Evaluation of Genomic Applications in Practice and Prevention (EGAPP)
VA Technology Assessment Program
BlueCross BlueShield Technology Evaluation Center
• Collaboration with Kaiser Permanente
ECRI Institute
Drug Evaluation Review Project (OHSU and RTI-UNC EPCs)
Hayes, Inc.
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Looking to Evidence Experts - International
Collaborations
• Cochrane Collaboration and Library
• International Network of Agencies for Health Technology Assessment
• European network for Health Technology Assessment (EUnetHTA)
• EuroScan
• Health Technology Assessment International (HTAi)
• International Society for Pharmacoeconomics and Outcomes Research
(ISPOR)
Agencies
• Europe - UK: NICE, NETSCC HTA, NHSC; Germany: IQWiG,
DAHTA@DIMDI; France: CEDIT, HAS; Spain: AETS, CAHTA;
Netherlands: GR, CVZ, ZonMw; Sweden: SBU, TLV; Denmark:
DACEHTA; Finland: FinOHTA; Belgium: KCE; Poland: AHTAPol
• Canada: CADTH (CDR), AETMIS
• Australia: PBAC, MSAC; Brazil: DECIT-CEGATS, Anvisa; S. Korea:
HIRA; Taiwan: DHTA
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What’s Next? Horizon Scanning
•
•
EuroScan (INAHTA members; secretariat at NHSC, UK)
National Horizon Scanning Centre (NHSC, UK)
•
Australia & New Zealand Horizon Scanning Network
•
Canadian Agency for Drugs and Technologies in Health
(CADTH) Horizon Scanning Service
•
ECRI Emerging Technology Services (USA and Europe)
 TARGET (TA Resource Guide for Emerging Technologies)
 Health Technology Forecast / Health Technology Trends
•
CMS Council on Technology and Innovation
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“Managed Entry” Taxonomy
Source: Carlson et al., 2009, under review
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Managed Entry Arrangements by Type
Scheme type
N
CED: Only
In research
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Product
Data collection Explicit link between Eval. of treatment
types
to inform popul. reimb. level and
response as condition
covered
decision-making product performance for coverage
Devices,
Pharm.
(off label)
Yes
No
No
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Devices,
Pharm.
(on label)
Yes
No
No
9
Pharm.
(on label)
No
No
Yes
PLR: Outcomes
guarantee
10
Pharm.
(on label)
Yes (42%)/No
Yes
No
PLR: Pattern or
process of care
1
Devices
Yes
Yes
No
CED: Only with
research
Conditional treatment
continuation
Total
54
CED: Coverage with evidence development
PLR: Performance-linked reimbursement
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Source: Carlson J, Sullivan SD, Garrison LP, Neumann PJ,
Veenstra DL. Pay for performance: A taxonomy and evaluation of
performance-based reimbursement schemes between healthcare
payers and manufacturers. 2009; Under Review.
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In Search of Evidence on …
•
•
•
Comparisons to standards of care
Specific indications
Health outcomes
 Even for screening and diagnostic tests and imaging
 If not direct evidence, then indirect
•
•
•
•
•
Clinically meaningful follow-up/duration
Relevance to beneficiary populations
Real-world settings
Subgroups, heterogeneity of treatment effects (HTEs)
RCTs (or PCTs) still gold standard
 Not always appropriate or necessary
 What’s the best you have?
The Lewin Group
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National Health Policy Conference
Using Evidence to Design Benefits
February 9, 2010
Washington, DC
Clifford Goodman, Ph.D.
Vice President
The Lewin Group
Falls Church, Virginia USA 22042
clifford.goodman@lewin.com
The Lewin Group
DO NOT TRANSMIT OR REPRODUCE WITHOUT PERMISSION
23