Understanding the Pricing and Reimbursement Outcomes of Advanced Therapy Medicinal Products (ATMP) in Spain

OBJECTIVES: Assess the key factors influencing the pricing and reimbursement (P&R) outcomes of Advanced Therapy Medicinal Products (ATMPs) in Spain.

METHODS: Secondary research to analyse the ATMPs approved in Europe from October 2009 to February 2024, that entered the P&R process in Spain, and how different factors could impact drug availability and timeframes.

RESULTS: A total of 26 ATMPs were approved in Europe. Of these, 18 initiated the P&R process in Spain: 7 (47%) received a positive resolution and 8 (53%) received a negative resolution. Most of them were gene therapies (80%), orphans (80%), and authorised under exceptional circumstances or conditional approval (87%). The approval was based on phase I/II (47%) or phase III (40%) clinical trials, predominantly open-label (94%), non-comparative (87%), and non-randomized (80%). A huge proportion were antineoplastic/immunomodulatory agents (53%), evaluated with hard outcome measures (60%) as a first-line treatment (54%). Among the funded therapies, all of them were subject to price discounts (range: 4-38%), and 86% had restricted access to the funded indication and payment-by-results agreements. Comparisons between funded and non-funded therapies reveal the following points: a) All funded therapies had orphan designation, compared to 63% of non-funded therapies; b) all were approved based on phase I-III studies versus 75%; c) 86% were studied using a robust variable versus 38%; d) 57% demonstrated clinical benefit versus 25%; e) 43% were treatments for third-line or later (3L+) versus 13%. In addition, the average time from European approval to funding resolution was 21 months for funded therapies, compared to 27 months for non-funded therapies.

CONCLUSIONS: This analysis highlights the importance of making innovative therapies accessible while upholding stringent standards for clinical evidence and cost-effectiveness. The findings indicate that improving the efficiency of the approval process and implementing flexible reimbursement models could enhance patient access to advanced therapies in Spain.