pharmacoeconomic assessment through market approval and beyond: theory and operations
TRANSCRIPT
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Welcome
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Agenda for Today’s Webinar
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o Overview of pharmacoeconomic (PE) assessmento Data collection for PE assessment o PE assessment at different phaseso Implementing data collection at different phaseso Collection of real world PE datao Future considerationso Q&A
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Overview of PE Assessment
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Why PE Assessment?o Need for product differentiation in competitive
marketo Challenging reimbursement requirementso Blend scientific and marketing objectives to help
sponsors tell value story of their productso Maximize potential return on investment
Physician Led | Therapeutically FocusedCopyright© 2015
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Main Types of PE Assessmento Cost-minimization analysiso Cost-effectiveness analysiso Cost-utility analysiso Cost-benefit analysiso Budget impact modeling
Physician Led | Therapeutically FocusedCopyright© 2015
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Cost-Minimization Analysis (CMA)
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o Compare costs of two or more drugs or therapies to determine least costly option
o Baseline costs will always include acquisition costs for drugs or therapies under consideration as well as costs of preparation and administration
o Additional costs considered depend on perspective of analysis
o Main benefit: cheaper and easier than cost-effectiveness analysis
o Main drawback: assumption that two drugs or therapies are used at equivalently effective doses
Physician Led | Therapeutically FocusedCopyright© 2015
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Cost-Effectiveness Analysis (CEA)o More comprehensive than CMA in that it
considers both costs and effectiveness of two or more drugs or therapies
o Costs measured in monetary units of interest (Eg, $, £, €)
o Effectiveness generally measured using one or more clinical outcome Examples:
• Years of life saved• Hospitalizations averted• Complications prevented
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Incremental Cost-Effectiveness Ratio (ICER)o ICER is main outcome of CEAo ICER calculated as: (∆ Costs / ∆ Effectiveness)
Examples:• Incremental cost per life year saved• Incremental cost per hospitalization averted• Incremental cost per complication prevented
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Willingness to Pay Thresholdo ICER is then compared against one or more
willingness-to-pay thresholds to determine if drug therapy of interest is cost-effective, cost-saving, or cost-neutral
o Can be plotted on cost-effectiveness planeo Multiple comparisons can be conducted using
cost-effectiveness acceptability curve (CEAC)
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Cost-Effectiveness Plane
∆ Co
sts
∆ Effectiveness
Maximum Acceptable
ICER
Accept
Reject
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Cost-Effectiveness Acceptability CurvePr
obab
ility
Cost
-Eff
ectiv
e
Threshold Cost-Effectiveness RatioPhysician Led | Therapeutically Focused
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Cost-Utility Analysis (CUA)o CEA that accounts for quality of time gained or
losto Quality of life (QOL) measured using utility
Generally ranges from 0 (dead) to 1 (perfect QOL) For some conditions (Eg, advanced ALS), negative
utility has been considered
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Quality-Adjusted Life Years (QALY)o Most common ICER assessed in CUA is cost per
QALY gained QOL increase of 0.5 to 0.75 for 4 years = 1 QALY
gainedo Depending on condition or therapy, other
timeframes can be used (Eg, month or day)
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Cost-Benefit Analysis (CBA)o Differs from CEA in that effectiveness is also
monetized Eg, year of life saved could be equated to year of
productive value to society, measured as per capita GDP or average annual income
o Cost-benefit ratio calculated as:(∆ Costs / ∆ Monetized Value of
Effectiveness)
Physician Led | Therapeutically FocusedCopyright© 2015
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Budget Impact Modeling (BIM)o CMA, CEA, CUA, and CBA often targeted toward
broader audienceso BIM generally designed for specific audiences,
particularly third party payerso Measures net cumulative treatment cost with drug
or therapy of interest for specified number of patients in particular population
o Impact of particular drug or therapy is assessed as effect on cost per member per month (PMPM)
Physician Led | Therapeutically FocusedCopyright© 2015
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Perspective of PE Assessmento Who will pay for drug or therapy and who will
benefit?o Potential perspectives include:
Healthcare provider Third party payer Patient/Caregiver Society
o Different perspectives warrant different considerations
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Time in Context of PE Assessmento Timeframe of disease (acute vs. chronic) and
therapy will determine time horizon for PE assessment
o When in development cycle should PE assessment be initiated and how long should it continue? Contemporaneous with later Phase III is common
but can consider earlier Phase III or Phase II Collection of real world PE data?
Physician Led | Therapeutically FocusedCopyright© 2015
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Data Collection for PE Assessment
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o Clinical Data Prospective Retrospective
o Claims Data Standalone Link with clinical data: Carefully Consider
• Consent• HIPAA• Blinding
Data Sources – Clinical and Claims
=
PayerTrial
Payer Trial
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Cost Datao Primary
CMS 1450 (UB04 Uniform Bill) CMS 1500 (Claim Form)
o Secondary CPT (AMA’s Current Procedural Terminology), other codes mapped to costs from payers RED BOOK Published Reports
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Collecting and Integrating Data from Multiple Sourceso Augment eCRF
Additional form matching UB04/CMS 1500 Instruct site on finding UB04/CMS 1500 in other file Ask site to complete UB04/CMS 1500 from scratch
o Code collected data in-house using CPT, or other cost source
o Map MedDRA codes utilized as standard part of clinical trial to CPT, or other cost source
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PE Assessment at Different Phases
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Drug Development Cycle
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Different Phases to Considero Phase IIo Earlier phase IIIo Phase III piggyback studieso Peri-authorizationo Post-authorization / Real World
o Comparing and contrasting approaches for smaller/mid-size relative to larger sponsors Developing a plan that encompasses all relevant
phases
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Implementing Data Collection at
Different Phases
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Best Practices for Changing the eCRFo Include all stakeholders in the decision
Site feedback Statistical considerations Impact on regulatory submissions
o Plan and communicate cutover o Design database to be flexible
Dropdowns, normalized structureo Off-line development and validationo Cutover on weekend
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Collecting Real World PE Data
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Why Real World PE Data?o Differentiation in costly yet lucrative landscape
$2.6 billion to bring new drug to market (Tufts CCSD) Global sales (2014) for top 25 pharmaceutical
companies were $548 billion (GlobalData)o More robust data than literature-based modelso More relevant than earlier phase clinical datao Timing a potential issue
Prospective longer to conduct than ideal Retrospective later to collect than ideal
o True cost-effectiveness rather than cost-efficacy
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Are Payers the True Decision Makers?
o Roles of FDA and payers, including Medicare and Medicaid, are constantly evolving
o Drug approval becoming “easier” (Forbes) 2008: 50% of new molecular entities (NMEs)
approved by FDA 2014: 88% of NMEs approved
o US District Court ruling re: Amarin’s Vascepa®
o Increased emphasis on generation and dissemination of evidence aimed at payers Real World Evidence/Outcomes liaisons at larger
sponsors
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Real World vs. Earlier PE Assessment: Functional Considerations
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Study Activity Real World Earlier PhaseStudy feasibility and document collection
More likely remote More likely in-person
Site qualification and initiation
More likely remote More likely in-person
Routine monitoring visits • Less frequent• Data-driven
• More frequent• Involves assessment
of protocol adherenceSource data verification More targeted Broader
Data review More centralized More dispersedCost data sources Targeted claims
databasesVarious sources
Effectiveness data sources Targeted EHR Various sources
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Target Data for Hybrid EHR Sourcing
Problem data for EHR systems
Stop datesScaled data
Surveys/PROsCosts
Better data for EHR systemsCoded for paymentsAlready transactional (lab systems, pharmacy)
Most problematic data for sitesHigh volumeComplexQuery-prone
Research vs.Healthcare
LabsMeds
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Recruitment and Retention Considerationso Well-integrated EDC, EHR, and ePRO will
enhance retention of sites and patientso Recruitment population for real world studies
differs from population for RCTso Real world studies tend to be longer, which has a
direct impact on site motivation and patient retention
o Gauging and developing site experience and motivation are essential in optimizing enrollment
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Site Training Considerationso Establishing study expectations from the outset is
essentialo Sites and Investigators:
How are patient records structured?• How find UB04, cost data?
Capacity for integration with EDC? Research-naïve?
• Real world and/or PE research experience?• Clinical trial experience?
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Regulatory and Safety Considerations
o Regulatory requirements for non-interventional studies are evolving and dependent on locality or localities in which study conducted
o Emphasis on scientific credibility and data protectiono Good Pharmacoepidemiology Practice (GPP)
proposes minimum practices and procedures to ensure data quality, integrity, and adequate documentation
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Future Considerations
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Future Considerationso PE assessment in earlier and later phases,
including real world, are important supplements to clinical trials
o Development cycle does not end with approvalo 51% of sponsors think in-house and outsourced
heath economics and outcomes research (HEOR) studies will increase significantly or moderately by 2018 (Industry Standard Research, 2014)
o Flexibility is essential
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Thank You
Lee WalkeVice President e-Clinical
Matthew J. Page, Ph.D., M.P.P.Epidemiologist
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Q&A