Analysis of Funding Conditions for Orphan Medicines in Spain: Trends in Pricing & Reimbursement

OBJECTIVES: As well as confidential discounts, pharmaceuticals in Spain are often subject to additional funding conditions, with the online platform Seguimed used to monitor sales and consumption. Funding conditions include a restricted population, expenditure ceilings, payment by results agreements, and trial of drug at no cost. The objective of this analysis is to explore how use of funding conditions has developed over the past three years, specifically for orphan medicines.

METHODS: All orphan medicines that achieved reimbursement in Spain between 1^st January 2021 and 11^th June 2024 were included in the analysis. The Spanish information system BIFIMED was used to extract data on funding conditions for each medicine. The data was then analysed over time to explore trends emerging in the use of funding conditions to manage drug spending.

RESULTS: In total, 59 orphan drugs achieved reimbursement in Spain between 01/01/2021 and 11/06/2024. On average, each drug was subject to at least 2 funding conditions (mean 2.1). Common funding conditions included: restrictions on eligible population (47.5%), maximum cost per patient (25.4%), and early access with the commitment to pay a rebate based on agreed price (23.7%). Notably, our analysis suggests use of clinical protocols and Valtarmed have reduced over time. On the other hand, use of early access, with a commitment to pay a rebate based on the ultimately agreed price, and trial of drug at no cost to establish responder status, have increased.

CONCLUSIONS: Funding conditions remain a commonly used tool to manage spending on pharmaceuticals in Spain. Our analysis shows that almost half of orphan medicines are reimbursed in a restricted population. In addition, an increase in the use of early access with the commitment to pay a rebate based on agreed price suggests Spanish payers are trying to speed up time to access.