Preparing Health Technology Submissions for Pharmaceutical Products

Content

• Executive Summary
• Background
• Overview
• Chapter 1: Global Formulary Submission Requirements
  • Chapter outline
  • 1.1 Introduction
  • 1.1.1 Key documents
  • 1.1.2 The second level
  • 1.1.3 Health technology assessments (HTAs)
  • 1.1.4 The emergence of formulary submission guidelines
  • 1.2 Current formulary submission standards
  • 1.2.1 PBAC: standards for clinical assessment
  • Case study 1.1: The PBAC guidelines
  • 1.2.2 England and Wales, NICE: standards for modeled cost-effectiveness claims
  • Case study 1.2: The NICE guidelines
  • 1.2.3 WellPoint: standards for monitoring and validating claims
  • Case study 1.3: The WellPoint guidelines
  • 1.2.4 The Scottish Medicines Consortium
  • Case study 1.4: The SMC guidelines
  • 1.2.5 US: AMCP - an interim standard
  • Case study 1.5: The AMCP guidelines
  • 1.2.6 Process and dossier submissions
  • Case study 1.6: Identifying reimburser requirements
  • 1.2.7 Transparency and process
  • 1.3 Hierarchy of clinical evidence
  • 1.4 Formulary recommendations and assignments
  • 1.5 The role of guidelines
  • Case study 1.7: The future of NICE - what could be NICER?
  • 1.6 Linking cost-effectiveness and budget-impact claims
  • Case study 1.8: Viagra versus the PBAC
  • 1.7 Overview: managing patient populations
  • Notes
• Chapter 2: Guidelines from a Global Perspective
  • Chapter outline
  • 2.1 A global guideline overview
  • Case study 2.1: The ISPOR guidelines summary
  • 2.2 Formulary submission guidelines: documentation and process
  • 2.3 Health technology assessments (HTAs) and the life cycle of a drug
  • 2.4 Disease area and therapeutic class reviews
  • 2.5 Bias and compliance
  • 2.6 Technology scoping
  • 2.7 The global dossier: meeting evidentiary and analytical standards
  • 2.2: Proposed outline for a global dossier
  • Notes
• Chapter 3: Uncertainty - Net Benefits, Product Ranking and the Reference Case
  • Chapter outline
  • 3.1 Uncertainty in cost-effectiveness claims
  • 3.2 Ranking therapy interventions
  • 3.3 ICERs and net benefit measures
  • 3.4 Defining net benefits
  • 3.5 Interpreting ICERs
  • 3.6 Net monetary benefit
  • 3.7 Probabilistic sensitivity analysis
  • 3.8 Estimating cost-effectiveness acceptability curves
  • Case study 3.1: Modelling a probabilistic sensitivity analysis
  • 3.9 Interpreting, monitoring and validating claims
  • 3.10 The NICE reference case
  • Case study 3.2: NICE reference case requirements
  • Case study 3.3: The EQ-5D and the SF-6D in liver transplant patients
  • 3.11 Implications of the reference case requirements
  • 3.12 Overview: thresholds and evidentiary standards
  • Notes
• Chapter 4: The Clinical Outcomes Case
  • Chapter overview
  • 4.1 Literature searches
  • 4.1.1 Key databases
  • 4.1.2 Reference inclusion/exclusion criteria
  • Case study 4.1: PBAC requirements for literature searches
  • 4.2 Bias and systematic reviews
  • 4.2.1 Randomisation
  • 4.2.2 Follow-up
  • 4.2.3 Blinding
  • Case study 4.2: Bias assessment in clinical trials
  • 4.2.4 Filtering studies
  • 4.3 Hierarchies of clinical evidence
  • Case study 4.3: The PBAC and WellPoint hierarchies of clinical evidence
  • 4.4 Summarising clinical studies
  • Case study 4.4: Meeting PBAC trial summary requirements
  • 4.5 Quality-scoring clinical studies
  • Case study 4.5: The Jadad quality-scoring algorithm
  • 4.6 Pooled clinical data and meta-analyses
  • Case study 4.6: The PBAC requirements for meta-analysis
  • 4.6.1 Identifying relevant studies
  • 4.6.2 Eligibility criteria
  • 4.6.3 Abstracting data
  • 4.6.4 Statistical models
  • 4.7 Adverse events and side-effect profiles
  • Case study 4.7: Pharmacoepidemiology
  • 4.8 Defining comparator products 4
  • Case study 4.8: Comparator therapies in the PBAC guidelines
  • 4.9 Epidemiology
  • Case study 4.9: WellPoint epidemiology profiling requirements
  • 4.10 Place of product in therapy
  • Case study 4.10: The PBAC and expert opinion
  • 4.11 Product profile
  • Case study 4.11: WellPoint product profile requirements
  • 4.12 Therapy intervention strategies
  • Case study 4.12: NICE recommendations for Relenza in the treatment of influenza
  • 4.13 Linking meta-analyses to modelled claims
  • Case study 4.13: Defining clinical parameters for cost-effectiveness modelling
  • 4.14 Monitoring and validating clinical claims
  • Case study 4.14: The NICE appraisal of beta interferon and glatiramer for multiple sclerosis
  • Notes
• Chapter 5: The Health Economics Case I - Generating Modelled Cost-effectiveness Claims
  • Chapter outline
  • 5.1 Types of modelled claim
  • Case study 5.1: Modeling criteria in the PBAC guidelines
  • 5.2 Decision-model frameworks
  • 5.3 Resource units and direct costs
  • Case study 5.2: Current procedure terminology (CPT) codes
  • 5.4 Valuing resource units
  • 5.5 Indirect costs
  • Case study 5.3: Demonstrating workplace productivity benefits
  • 5.6 Measuring outcomes
  • 5.6.1 Construct
  • 5.7 Modelling, sensitivity and simulation analyses
  • 5.8 Spreadsheet models
  • 5.9 Monitoring and validating cost-outcome claims
  • Case study 5.4: The impact of inhaler type on monthly treatment costs of asthma - a retrospective study
  • 5.10 Meta-models
  • Case Study 5.5: The CORE diabetes meta-model
  • Notes
• Chapter 6: The Health Economics Case II - Estimating System Impacts
  • Chapter outline
  • 6.1 Defining terms
  • 6.2 Forecasting product uptake
  • Case study 6.1: SMC requirements for product uptake projections
  • 6.3 Patient switching and target populations
  • 6.3.1 Defining a target population
  • 6.3.2 Market segmentation
  • 6.4 Budget-impact claims
  • 6.4.1 Resource units and unit pricing
  • 6.5 Estimated pharmacy budget impact
  • 6.6 Estimated medical budget impact
  • 6.7 Estimated total budget impact
  • Case study 6.2: PBAC requirements for financial impact assessment
  • Note
• Chapter 7: Responding to Disease Area and Therapeutic Class Reviews
  • Chapter outline
  • 7.1 Life-cycle product assessment
  • 7.1.1 Clinical assessments
  • 7.1.2 Anticipating requests for monitoring and validation
  • 7.2 Assessing claims
  • 7.3 Contractual requirements
  • 7.4 Experimental approaches: naturalistic trial designs
  • Case study 7.1: The role of naturalistic trials
  • 7.5 Non-experimental designs
  • 7.5.1 Case-control studies
  • 7.5.2 Cohort studies
  • 7.6 Practice pattern variations
  • Case study 7.2: The WellPoint agenda
  • Notes
• Chapter 8: Summary and Conclusions
  • Chapter outline
  • 8.1 The future of technology appraisals
  • 8.2 Technology appraisals in the short term
  • 8.3 Technology appraisals in the longer term
• Glossary
• List of Figures
  • Figure 3.1 Benefit and willingness to pay
  • Figure 3.2 Cost-effectiveness plane
  • Figure 3.3 Net monetary benefit
  • Figure 3.4 Ranking net monetary benefits
  • Figure 3.5 Cost-effectiveness acceptability curve
  • Figure 3.6 Decision model: Therapy A versus Therapy B
  • Figure 3.7 Simulated distribution of differences in costs
  • Figure 3.8 Simulated distribution of differences in outcomes
  • Figure 3.9 Distribution of cost and outcome difference coordinates in the cost-effectiveness plane
  • Figure 3.10 Simulated cost-effectiveness acceptability curve
• List of Tables
  • Table 2.1 Key formulary submission guidelines: documentation and process
  • Table 3.1 Parameter values: Therapies A, B and C
  • Table 3.2 Simulation pairs of cost and outcome differences
  • Table 3.3 Simulated proportion of coordinate cost and outcome difference by willingness-to-pay threshold
  • Table 4.1 Grading of clinical studies