High-impact medicines, system sustainability and the role of access management: evidence from Catalonia

Abstract

Background The reimbursement of high-impact medicines (HIMs) poses growing clinical and budgetary challenges for publicly funded health systems. In Catalonia, pharmaceutical spending has increased considerably in recent years, driven by the uptake of high-cost therapies and expanded indications under clinical and economic uncertainty. To mitigate these risks, payers have increasingly used managed access mechanisms in reimbursement strategies. Objective To assess the economic impact of newly reimbursed high-impact medicines in Catalonia and describe the role and economic implications of managed access mechanisms applied to address clinical and financial uncertainty. Methods We conducted a retrospective study using real-world data from Catalonia's health service registries and billing systems. Expenditure trends for HIMs between 2019 and 2024 were analysed. Newly reimbursed indications from 2022 to 2024 were evaluated by therapeutic class, volume of patients, and aggregated expenditure per patient, taking into account the managed access mechanisms applied. Results From 2019 to 2024, HIM expenditure rose by 53% in nominal terms, from €1.10 billion to €1.69 billion; after adjustment for inflation, 2019 expenditure corresponded to approximately €1.30 billion in 2024 euros, indicating a real increase of approximately 30%. In 2024, HIM expenditure represented about 11% of the total healthcare expenditure managed by the Catalan Health Service. The top 10 medicines accounted for 43% of expenditure. Newly reimbursed indications in 2024 represented €23.6 million. Managed access mechanisms were applied to nearly three-quarters of these indications, including indication-based restrictions and formal agreements. Formal managed access agreements included confidential discounts, spending caps, patient-level spending caps, free-dose schemes, price–volume agreements, outcome-based payment models, and staggered payments. Conclusions Newly reimbursed HIMs exert concentrated expenditure pressure. These findings illustrate how the broad use of managed access mechanisms can support balancing access to innovation with health system sustainability. Future research is needed to identify the most effective managed access strategies to facilitate access to new HIMs at costs sustainable for health systems.