Health Economics from Theory to Practice: Optimally Informing Joint Decisions of Research, Reimburse

OverviewAn Appropriate Underlying Objective FunctionPrinciples for Constrained Optimisation Across Health Promotion, Prevention and Care SettingsOverview of ChaptersReferencesI Principles and Practice for Robust Net Benefit Analysis Informing Optimal Reimbursement (Adoption and Financing) Decisions Across Individual and Community-Focused Programs Using Trial, Model and Network Multiplier MethodsPrinciples and Practice for Trial-Based Health Economic AnalysisOverviewPrinciples for Robust Health Technology AssessmentDecision Analytic Approaches to Robust AnalysisWhy Use Incremental Net Benefit and Not Incremental Cost Effectiveness RatiosIllustrating Principles Within Study: The LIPID Trial Case StudyRepresenting Cost Effectiveness UncertaintyBootstrapping the CE DistributionFieller’s MethodUseful Cost Effectiveness Summary Measures from Bivariate Distributions Conditioning on Threshold Values for EffectHow Should Economically Meaningful Threshold Values for Effects Be Estimated?ConclusionDiscussion - Satisfying Coverage, the Need for Robust Evidence Synthesis, Translation and ExtrapolationReferencesAvoiding Frankenstein’s Monster and Partial Analysis Problems: Robustly Synthesising, Translating and Extrapolating EvidenceIntroductionSetting the Scene: Frankenstein’s Monster or the Vampire of TrialsCost Minimisation and the Absence of Evidence FallaciesIndirect Comparison and Avoiding Framing Biases with Relative Risk in Evidence Synthesis of Binary OutcomesWhat Causes Reversal of Treatment Effect with Relative Risk?Preventing Framing Biases in Evidence TranslationDoes Relative Risk Consistently Estimate Absolute Risk Difference in Translating Evidence with Alternate Framing of Binary Events?Does the Odds Ratio Allow Consistent Estimation of Absolute Risk Difference in Translating Trial Evidence to Jurisdictions of Interest?Extrapolating Cost Effectiveness Evidence Beyond Trial Duration for a Jurisdiction of InterestReferencesBeyond the Individual: Evaluating Community-Based Health Promotion and Prevention Strategies and Palliative Care Domains of Effect Simon Eckermann and Nicola McCaffreyIntroductionEvaluating Health Promotion and Prevention: Moving Beyond Individual Measures Within StudyThe Stephanie Alexander Evaluation Case StudyConclusion: Health Promotion and Prevention in Complex Community SettingsPalliative CareEconomic Evaluation in Palliative CareReferencesII Joint Research and Reimbursement Questions, Optimising Local and Global Trial Design and Decision Making Under Uncertainty Within and Across Jurisdictions with Value of Information MethodsThe Value of Value of Information Methods to Decision-Making: What VOI Measures Enable Optimising Joint Research and Reimbursement Decisions Within a Jurisdiction?Expected Value of Information Principles and MethodsTaking Occam’s Razor to VOI Methods - What Is Necessary and Sufficient to Address Research Questions?Candidate Set 1: Per Patient and Population EVPICandidate Set 2: Per Patient and Population EVSICandidate 3: The Expected Value Less Cost or Expected Net Gain (ENG) of a Given TrialWhat Is Required to Inform Decision-Making Questions: Optimising ENG or Return on ResearchBroader Dangers of Population EVPI in Allocating Research FundingWhat VOI Method(s) Enable ENG OptimisationAppropriately Allowingfor Within Jurisdiction Decision Contexts Applying the CLTHow Can VOI Methods Inform the Choice Between AN, DT and AT Where Feasible?Expected Value and Cost of Trials with a Delayed Reimbursement Decision (DT Versus AN)EVSI where Adopting and Trialing is FeasibleIllustrating Optimising of Joint Optimising Research and Reimbursement Decisions - Early Versus Late External Cephalic VersionComparing AT, AN and DTDistinguishing Between Costs of Adoption, Delay and ReversalMore General Implications for Optimising Joint Research and Reimbursement DecisionsVOI Advantages over Frequentist Designs in Enabling Efficient Research Design for Joint Research and Reimbursement DecisionsConclusion and Discussion of Broader VOI Methods Issues Arising for Decision-Making Within JurisdictionReferencesGlobally Optimal Societal Decision Maker TrialsIntroductionExpected Value and Costs Across Jurisdictions for Global Trial DesignIllustrating Methods: Globally Optimal Trial Design (The USA, UK and Australia)Explicitly Addressing Imperfect Translation in Optimal Global Trial DesignGlobal Trials for Existing TechnologyConclusion: Optimal Global Trial Design as First Best SolutionReferencesValue of Information, Pricing Under Uncertainty and Risk Sharing with Optimal Global Trial DesignIntroductionPricing Under UncertaintyIllustrating Threshold Pricing Under UncertaintyPricing Under Uncertainty with Adoption in a Global TrialCircuit Breaker Advantages in Bringing Societal Decision Maker and Manufacturer Interests Closer TogetherDeeper Implications for Implementation and PracticeBottom Line for VOI MethodsReferencesIII Regulating Strategies and Providers in Practice: The Net Benefit Correspondence Theorem Enabling Robust Comparison of Multiple Strategies, Outcomes and Provider Efficiency in Practice Consistent with Net Benefit MaximisationBest Informing Multiple Strategy Cost Effectiveness Analysis and Societal Decision Making: The Cost Disutility Plane and Expected Net Loss Curves and FrontiersAn Introduction to Multiple Strategy Comparison and Limitations of Fixed Comparator Two-Strategy Presentations and Summary MeasuresOvercoming Fixed Comparator Problems - Multiple Strategy Comparison of Costs and Effects with Flexible Axes on the C-DU PlaneNet Loss Statistics, Expected Net Loss Curves and the Expected Net Loss FrontierThe ENL Frontier and EVPIBest Presentation and CE Summary Measures to Inform Risk-Neutral or Somewhat Risk-Averse Societal Decision Making with Two and More than Two StrategiesDiscussion of the CEA FrontierConclusionReferencesIncluding Quality of Care in Efficiency Measures: Creating Incentives Consistent with Maximising Net Benefit in PracticeOverviewThe Need to Include Quality in Efficiency Measures Consistent with Maximising Net BenefitThe Quality of Care ChallengeNBCT ProofPolicy Implications of the NBCT FrameworkFurther ExtensionsReferencesMultiple Effects Cost-Effectiveness Analysis in Cost-Disutility SpaceIntroductionExtending Cost-Effectiveness Analysis on the Cost-Disutility PlaneTechnical Efficiency FrontierDeterministic AnalysesThreshold Regions Across Effect Values where Strategies are OptimalSummary Measures Under Uncertainty: The Value of Accounting for Joint UncertaintyExpected Net Loss (ENL) and ENL PlanesExpected Net Loss ContourCost-Effectiveness Acceptability PlanesMultiple Domain Palliative Care ExampleMethodsModel StructureParameters EffectivenessAnalysisResultsConventional AnalysesComparison in Cost-Disutility SpaceDeterministic AnalysesStochastic AnalysisDiscussionConclusionReferencesIV The Health Shadow Price and Other Key Political Economy and Policy Issues: Appropriate Threshold Pricing and Policy Application of Methods for Optimising Community Net Benefit with Budget ConstraintsThe Health Shadow Price and Economically Meaningful Threshold ValuesOverviewWhy Are Economically Meaningful Threshold Values CriticalHistorical Threshold Values and Opportunity CostsConsidering Displaced Services as a Threshold: The Straw Man Outside the RoomDistinct Dangers of Using Displaced Service Thresholds Over Time, Whether Assumed or Actual and Applied Inconsistently or ConsistentlyThe Health Shadow Price for Reimbursement (Adoption and Financing)The Health Shadow Price and PBMA as a Pathway to Allocative EfficiencyHealth Shadow Prices for Cost Saving Investment OptionsConclusionReferencesPolicy Implications and Applications Across Health and Aged Care Reform with Baby Boomer Ageing - from Age and Dementia Friendly Communities to Palliative CareIntroductionHealth-Care Policy for Successful Ageing: Where Should Health and Aged Care Reform Be Heading (The Importance of Dementia and Age-Friendly Community Environments)How Can Health Economics Help: More Than Cost-Effectiveness AnalysisAgeing Expenditure Catastrophe: Prior Myths and Future ChallengesWhat Has Driven Real Health Expenditure Growth Rather Than Ageing?Ageing Reform Options in the CommunityDementia-Friendly Aged Care and Nursing Home DesignPalliative Care Reforms - Optimising Potential of Some Promising Low-Cost and Palliative Domain Supportive OptionsOptimising Medicinal Cannabis as an Effective, Low-Cost and Palliative Domain Supportive Programme OptionInternational Scientific, Trial and Practice EvidenceOpportunity Cost, Cost and Energy Use of Outdoor Versus Indoor CultivationOther Promising Palliative Preference Supportive Factor Priced Therapie for Delerium and Cancer CareBridging the Silos: Funding for Budget-Constrained Optimal Quality of CareWhat Funding Mechanism Provides Appropriate Accountability for Quality?Fundingfor Net Benefit Maximising IncentivesAgeing Policy ConclusionsHealth Economic Tools Aiding Health Reform Gets ThereReferencesConclusion
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