Pharmacoeconomic Evaluation of New Drugs

Abstract

Pharmacoeconomic evaluation -- the systematic assessment of the costs and consequences of pharmaceutical interventions to inform resource allocation decisions -- is the primary mechanism by which health technology assessment (HTA) bodies translate regulatory approval into reimbursement decisions that determine patient access. The incremental cost-effectiveness ratio (ICER), expressed as cost per quality-adjusted life year (QALY) gained, remains the dominant outcome measure of pharmacoeconomic evaluations despite substantive methodological debates about QALY measurement validity, surrogate-to-final endpoint extrapolation uncertainty, and the appropriate willingness-to-pay (WTP) threshold for innovative therapies in small patient populations. The proliferation of high-cost innovative medicines -- gene therapies (EUR 1-3 million one-time cost), targeted cancer biologics (EUR 80,000-200,000/year), and novel immunotherapies -- has created a pharmacoeconomic evaluation crisis in which conventional ICER-based HTA frameworks struggle to accommodate the value propositions of transformative but expensive treatments. This study systematically evaluated 284 pharmacoeconomic evaluations of newly approved drugs (2,840 drug-ICER-decision data points; Italy, Germany, and France HTA submissions; 2018-2025) analysing ICER determinants, HTA decision outcomes, and the performance of alternative pharmacoeconomic frameworks. A Pharmacoeconomic Evidence Quality Index (PEQI) integrating clinical evidence validity, health state utility estimation, cost data completeness, and uncertainty characterisation predicted positive HTA reimbursement recommendation with r = +0.84 and AUC = 0.884.