Advancing Health Economics, Services, Policy and Ethics

REAL WORLD EVIDENCE:RESEARCH APPROACHES TO HEALTH TECHNOLOGY MANAGEMENT

Stuart PeacockCancer Control Research, BC Cancer AgencyCanadian Centre for Applied Research in Cancer Control (ARCC)Simon Fraser University

• Single shot policy questions• Ongoing priority setting frameworks• Some points for discussion

Real world evidence and priority setting

• 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 quality-

adjusted 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

Prostate Cancer 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 quality-adjusted life years (QALYs)”

• PBMA is a practical framework to aid decision-makers 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

Program Budgeting and Marginal Analysis (PBMA)

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Real World Evidence and PBMADefine aim and

scope

Form Steering Committee

Determine current program budget

Establish decision-making criteria

Identify areas for resource

release

Identify areas for new

resource use

Make allocation recommendations

Validity check and final decisions

For each area identified:

Form Advisory Panel

Collect local costs/outcomes

Build Markov model - CUA

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

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• 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

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Study Sample – from HCP data871 women with BRCA1/2 test results in 2002-2007

203 confirmed BRCA1/2 mutation

positive 99 with no cancer (or no CAIS record of cancer)

105 BRCA1/2 positive cancer cases

87 patients with first cancer

668 mutation negative or uninformative

18 with other cancer or missing stage information

68 patients with complete records

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

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• Costs:– MRI screen: $277 (IH, BCCA and VIHA)– Bilateral mammography: $95 (2008 MSP)– Average diagnostic work-up: $187 (2008 MSP)

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

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Treatment Costs

In Situ Local Regional Distant

Surgery 3,394 3,365 3,595 3,057Chemo 33 3,625 9,108 5,753Radiation 0 3,785 10,909 6,835TOTAL 3,427 10,940 23,612 15,645

MR

Chemo 11,082Radiation 2,152Hospitalization 12,714TOTAL 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

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

Treatmentdecision

Diagnostictest

FLT3-ITD and NPM1 mutational testingICER=$65,186/LYG

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• Sustainability• Investments and disinvestments

• Personalized medicine – drugs• Personalized medicine - tests

Points for discussion

Advancing Health Economics, Services, Policy and Ethics

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