Pharmacoeconomics of high-cost therapies: real world challenges

INTRODUCTION: The emergence of high-cost therapies, particularly in oncology, rare diseases, and advanced therapy medicinal products, has increased the need for robust pharmacoeconomic evaluations to support healthcare decision-making. These innovative interventions often promise substantial clinical benefits but also pose challenges due to limited long-term data, high upfront costs, and uncertain cost-effectiveness. AREAS COVERED: This narrative review critically examines key pharmacoeconomic frameworks, e.g. cost-effectiveness analysis and cost-utility analysis, and highlights their limitations in assessing such novel therapies. The role of real-world evidence, advanced modeling techniques, and patient-reported outcomes is explored, alongside the ethical and policy considerations that arise in pricing, access, and reimbursement. Recent literature and case studies involving gene and cell therapies are reviewed, illustrating the complexity of determining value under uncertainty. EXPERT OPINION: The paper advocates for a more flexible, transparent, and patient-centered approach to health technology assessment, integrating societal values and dynamic reassessment mechanisms. Such strategies are essential for achieving sustainable access to high-value therapies while maintaining health system efficiency and equity.