Real world evidence and priority setting
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Transcript Real world evidence and priority setting
Stuart Peacock
Cancer Control Research, BC Cancer Agency
Canadian Centre for Applied Research in Cancer Control (ARCC)
Simon Fraser University
Advancing Health Economics,
Services, Policy and Ethics
• I have no conflicts of interest
Real world evidence and priority setting
• Single shot policy questions
• Ongoing priority setting frameworks
• Some points for discussion
Prostate Cancer Screening
• Prostate Cancer Screening policy: funded and led by ARCC
• Collaboration with ARCC, BCCA, Vancouver Prostate Centre (VPC),
and the Fred Hutchinson Cancer Research Centre
• We found that regular screening resulted in a loss of qualityadjusted life years, regardless of screening intensity, when quality
of life was factored into the model
• BCCA/VPC updated their 2012 provincial recommendation on PSA
screening to explicitly state that they did not support unselected,
population-based screening
“The incremental cost-effectiveness of
regular screening ranged from
$36,300/LYG, for screening every four
years from ages 55 to 69 years, to
$588,300/LYG, for screening every
two years from ages 40 to 74 years.
After utility adjustment, all screening
strategies resulted in a loss of qualityadjusted life years (QALYs)”
Program Budgeting and Marginal Analysis
(PBMA)
• PBMA is a practical framework to aid decisionmakers seeking to maximize benefits from scarce
resources
• Limitations of PBMA
– reliance on simple models
– perceived dependence on content expert’s subjective
estimates of effectiveness and/or benefits
– lack of comparability between measures of
effectiveness
6
Real World Evidence and PBMA
Define aim and
scope
Determine current
program budget
Form Steering
Committee
For each area identified:
Establish decisionmaking criteria
Identify areas
for new
resource use
Identify areas
for resource
release
Form Advisory
Panel
Collect local
costs/outcomes
Make allocation
recommendations
Build Markov
model - CUA
Validity check and
final decisions
MCDA Models
5 areas identified:
• Adjuvant trastuzumab in
breast cancer
• Bevacizumab in metastatic
colorectal cancer
• Mammography for women
with dense breast tissue
• PET for lung cancer staging
• MRI for breast cancer
screening
7
• Objective:
– Examine the cost effectiveness of MRI and mammography for
breast cancer screening in BRCA1/2 mutation carriers
• Current practice:
– 6 mo. alternating MRI and mammography for confirmed
BRCA1/2 carriers (& family)
– Annual mammography for others at high hereditary risk
• Rationale:
– MRI is more sensitive than mammography (75% vs. 32%) but
less specific (96.1% vs. 98.5%) and more expensive
Markov Model Design
9
Study Sample – from HCP data
871 women with BRCA1/2
test results in 2002-2007
203 confirmed
BRCA1/2 mutation
positive
105 BRCA1/2 positive
cancer cases
87 patients with first
cancer
68 patients with
complete records
668 mutation
negative or
uninformative
99 with no cancer (or no
CAIS record of cancer)
18 with other cancer or
missing stage
information
19 patients
diagnosed before
1995
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Data Sources for Model
Model Input
Sources
Cancer Incidence
Literature (meta-analysis)
Screening Sensitivity and
Specificity
Literature (meta-analysis)
Cancer Survival
BCCA Surveillance and Outcomes
data
Treatment procedures
BCCA records for BRCA1/2
population
Treatment Costs
BCCA Pharmacy, Radiation
Therapy and Administration; BC
Medical Services Commission
Utilities
Literature
11
Screening and Diagnostics
Sensitivity
Specificity
MRI
0.77
0.86
Mammography (in MRI arm)
0.39
0.95
MRI & Mammo (pooled)
0.94
0.77
Mammography
(Mammography
alone arm)
< 50 yrs
0.67
0.88
> 50 yrs
0.83
0.88
from meta-analysis by Warner 2008; Kerlikowske 2000
• Costs:
– MRI screen: $277 (IH, BCCA and VIHA)
– Bilateral mammography: $95 (2008 MSP)
– Average diagnostic work-up: $187 (2008 MSP)
12
Treatment Costs
In Situ
Local
Regional
Distant
Surgery
3,394
3,365
3,595
3,057
Chemo
33
3,625
9,108
5,753
0
3,785
10,909
6,835
3,427
10,940
23,612
15,645
Radiation
TOTAL
MR
Chemo
Radiation
11,082
2,152
Hospitalization
12,714
TOTAL
26,704
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Utilities
• Derived from
published quality of
life studies
• Screening has ‘full
health’ utility (1.00)
State
Utility
Diagnostics
0.987
In situ
0.965
Local
0.860
Regional
0.675
Distant
0.380
Remission
0.965
MR
0.380
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Results
Other ICER Results
•
Screening Mammography
annual screening mammography for women with greater than 75%
mammographic breast density had an ICER range of $565,912/QALY
• PET/CT
PET for NSCLC staging: $10,932/LYG
PET for SPN diagnosis: $64,062/LYG
•
Adjuvant Trastuzumab for breast cancer
use of adjuvant trastuzumab saves approximately $1,200,000 from the
Systemic Therapy budget annually
projecting survival scenarios forward 28-years produced an ICER of
$13,095/QALY
• Bevacizumab for metastatic colorectal cancer
Introduction of bevacizumab associated with an ICER of $43,058/QALY
Cost-effectiveness of Personalized Medicine
FLT3-ITD and NPM1
mutational testing
ICER=$65,186/LYG
Treatment
decision
Diagnostic
test
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Points for discussion
• Sustainability
• Investments and disinvestments
• Personalized medicine – drugs
• Personalized medicine - tests
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Advancing Health Economics,
Services, Policy and Ethics