in search of equity, efficiency and impact in hta: the case for evaluation platform in copd (epic)...
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In search of equity, efficiency and impact in HTA: the case for Evaluation Platform in COPD
(EPIC)
Mohsen Sadatsafavi MD, PhDUniversity of British Columbia
2015.04.13
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Disclosures and Acknowledgements
• I am not aware of any actual or potential conflicts of interest
• Have received funding for this work– The Canadian Respiratory Research Network– Genome Canada
• Team– Core development team: Zafar Zafari & Amir Khakban– Co-investigators: Don Sin, J Mark FitzGerald, Stirling Bryan
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Outline
• The Need– COPD as an escalating public health challenge– Research community’s need for a framework to attach value to the
research pipeline
• Objectives– Overall & specific
• The approach– Whole Disease Modeling as the conceptual framework– Conceptualization -> Evidence synthesis ->Implementation– iKT
• Challenges & Rewards
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The Need COPD, undefeated!
Canadian Lung Association – 2010, Khakban et. al. under reivew
• The fourth common cause of death, to be the third by 2020
• The only common chronic disease whose burden is increasing
• Under-diagnosis epidemic: for every 2 diagnosed COPD patients, 3 remain undiagnosed
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The NeedThe Canadian Respiratory Research Network
• First CIHR-funded national respiratory network in Canada
• 13 research platforms (including Health Economics)
• Major focus on health policy and public health research on chronic airway diseases
• Burden and relevance of unidentified obstructive lung disease a major focus of the network
www.respiratoryresearchnetwork.ca/
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The NeedExplicit health economic studies & evaluations
• Environmental Health platform– Attributable burden of air pollution
• Population Health platform– Attributable burden of asthma-COPD overlap syndrome
• Biomarker Discovery platform– Cost-effectiveness and budget impact of
diagnostic and prognostic biomarkers• Health Services Research platform
– Cost-effectiveness of screening/case detection of COPD at community level
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The NeedOur response
One model to rule them all
Prevention (smoking session)
Environmental contribution
Early diagnosis (screening/case
finding/diagnostic biomarkers)
Early intervention (CEA,EVI)
Optimal pharmacotherapy
Biomarkers for exacerbation
Optimal treatment of exacerbations
Readmissions
Self management
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Evaluation Platform in COPDObjectives
• Specific objective– To evaluate COPD case detection strategies (in
different at-risk subgroups) in terms of epidemiological consequences, cost-effectiveness, and budget impact.
• General objective– To create the first Canadian outcomes model of
COPD to support policy, practice, and research.
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The ProcessConceptual framework
• Whole Disease Modeling 1: Modeling the complete natural history of COPD2: Capturing subgroups/pathways of care3: Transferability of decision node4: Enabling evaluation of disinvestment options– Individual-level simulation
• Discrete Event Simulation– Metric for modeling natural history of disease
• FEV1, FVC– Open population
Tappenden et. al. Value in Health, 2012
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The Process
Conceptualizations
Evidence synthesis
Implementation
Calibration/Validation
Expert Advisory Committee
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The ProcessConceptualization
• Demographics– Agent creation, dynamic risk factors (e.g., weight)
• Smoking– Incidence, remission, and relapse
• Lung Function– Representing the core natural history component
• Co-morbidities– As important determinants of burden ofCOPD
• Payoffs– Costs, quality of life, life years, number of exacerbations
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The ProcessEvidence Synthesis
(A)
(B)
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0 1 2 3 4 5 6 7 8 9 10 11
FEV1
(L)
Time (years)
95% coverage interval
Mean FEV1 decline
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0.5
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FEV1
(L)
Time (years)
Chart Title
95% coverage interval
Mean FEV1 decline
FEV1(t)= FEV1(t0)+(t- t0).(β1.X1+β2.X2+ … +u1.Z1+ u 2.Z2+ …)+ε
Zafari et al, under review12
Web App:http://resp.med.ubc.ca/software/ipress/epic/fev1pred/
Mixed-effects regression using the Lung Health Study Data
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The ProcessImplementation
• Scalable platform, ready to accommodate future questions
• Enable PSA and even EVI– Require two-level Monte Carlo simulation
• Fast platform is required as the number of simulation runs will be enormous– Dedicated implementation platform
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The ProcessCalibration/Validation
Zafari et al, under review14
Internal validity External validity
LHS EUROSCOPE
Plans:- An external study for validation of exacerbation equations- Validation of model outputs against BC administrative health data
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Integrated Knowledge Translation
• Require constant engagement of– Expert to supply the ‘science’ behind the model– Stakeholders to navigate overall development process– Patients to prioritize outcomes, characterize real-world
experience of care• To be impactful, the product should be
– Available to all relevant stakeholders– Transparent in structure and assumptions
Careful documentation of structure and associations Interactive, free to use, Web Interface (iPRESS)http://resp.med.ubc.ca/software/ipress/epic/
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Challenges and Rewards• Soliciting clinical input in a meaningful way difficult• Significant time and resources required, in the era of
publish yet still perish• Is it possible to have a model that only needs to be
‘tweaked’ for new evaluations?• Sustainability?• Conceptualization forces us to think -> better
understanding of the disease process• Tremendous support (scientific and logistical)• CONSISTENCY• Transferability of methodology/technology
FacultyMohsen SadatsafaviJ Mark FitzGeraldStirling BryanLarry Lynd
Research StaffHamid TavakoliTania ConteRoxanne RousseauAmir Khakban
StudentsZafar ZafariWenjia Chen
Thank You!
Respiratory Evaluation Sciences Program (resp.med.ubc.ca)
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MethodsOther challenges
Discovery Proof of efficacy
Proof of efficiency
Submission to CADTH
Discovery Proof of efficacy
Proof of efficiency
I have a drug that works, and is good for CanadaShow me the proof
I have a drug that works, and is good for CanadaShow me it works, and I will let you know if we think it is good for Canada
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MethodsWhy is it relevant?
• Efficacy is discovery, efficiency is value demonstration
• The former is a duty of the innovator Government agencies as Resource Stewarts need to own the latter
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MethodsiKT
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The ProcessEvidence Synthesis
• FEV1 as the hallmark of COPD progression
• Previous focus on population-average, need for original data analysis to inform DES