Training Course on Health Economics of Pharmaceuticals

Module 1: Introduction to Health Economics and Pharmaceuticals
Fundamentals of Health Economics
Concept of scarcity, opportunity cost, and resource allocation in healthcare.
Efficiency vs. equity in pharmaceutical decision-making.
Pharmacoeconomics in Context
Role in clinical trials and post-marketing surveillance.
How pharmacoeconomics informs formulary decisions.
Types of Economic Evaluations in Pharmaceuticals
Cost-minimization: identifying least costly equivalent.
Cost-effectiveness analysis (CEA): comparing cost per health outcome.
Cost-utility analysis (CUA): QALYs and DALYs.
Cost-benefit analysis (CBA): monetizing health outcomes.
Pharmaceutical Market Dynamics
Patent protection and exclusivity.
Global trends in pharmaceutical expenditure.
Case Discussion: Comparing economic access to insulin in high-income vs. low- and middle-income countries.

Module 2: Pharmaceutical Pricing and Market Access
Drug Development and Cost Structure
R&D phases: discovery, pre-clinical, clinical trials, regulatory approval.
Fixed vs. variable costs in drug development.
Pharmaceutical Pricing Strategies
Cost-plus vs. value-based pricing.
Reference pricing and external price benchmarking.
Differential (tiered) pricing across markets.
Market Access and Affordability
Barriers to entry: regulatory approval, intellectual property rights, competition.
Role of generic entry in reducing costs.
Health Technology Assessment (HTA)
Function of HTA bodies in drug approval (NICE, EMA, FDA, WHO).
Evidence requirements for reimbursement submissions.
Activity: Groups design a pricing and market access strategy for a new oncology drug entering both high-income and LMIC markets.

Module 3: Pharmacoeconomic Evaluation Methods
Core Evaluation Methods
Cost-effectiveness analysis (CEA): interpreting ICERs.
Cost-utility analysis (CUA): incorporating patient-reported outcomes.
Cost-benefit analysis (CBA): willingness-to-pay approaches.
Cost-minimization analysis (CMA): use cases.
Measuring Health Outcomes
DALYs, QALYs, and patient preference tools (EQ-5D, SF-36).
Clinical endpoints vs. surrogate endpoints in trials.
Modeling Techniques in Pharmacoeconomics
Decision tree models for short-term interventions.
Markov models for chronic diseases.
Budget impact analysis for new therapies.
Managing Uncertainty in Analysis
Sensitivity analysis (one-way, probabilistic).
Scenario testing for market entry and pricing.
Exercise: Teams calculate a cost-effectiveness ratio comparing a new vaccine to an existing one using hypothetical data.

Module 4: Reimbursement and Policy Perspectives
Role of Payers in Pharmaceuticals
Public health insurance schemes (Medicare, Medicaid, NHIF).
Private insurers and managed care organizations.
HTA Agency Frameworks
NICE (UK), CADTH (Canada), ICER (US), EUnetHTA (EU).
Thresholds for cost-effectiveness and affordability.
Reimbursement Pathways
Risk-sharing agreements and outcome-based contracts.
Managed entry agreements for expensive therapies.
Ethical and Equity Considerations
Orphan drugs and rare disease therapies.
Trade-offs in allocating limited budgets across populations.
Case Study: Policy challenges in reimbursing high-cost CAR-T cell therapy.

Module 5: Future Trends and Global Challenges in Pharmaceutical Economics
Generics and Biosimilars
Regulatory pathways for approval.
Economic impact on healthcare expenditure.
Innovations and High-Cost Therapies
Precision medicine and pharmacogenomics.
Gene therapies and cell-based treatments.
Global Health Priorities
Essential medicines list (WHO) and affordability challenges.
Antimicrobial resistance (AMR) and incentives for antibiotic development.
Access to medicines in pandemics and public health emergencies.
Digital Health in Pharmaceuticals
Digital therapeutics and reimbursement models.
Real-world evidence (RWE) and big data in HTA.
Communicating Pharmacoeconomic Evidence
Writing policy briefs for decision-makers.
Presenting evidence to clinicians and patient advocacy groups.
Capstone Project: Teams design a pharmacoeconomic evaluation and reimbursement strategy for a new therapy addressing a priority disease area (e.g.,
diabetes, cancer, rare disease)