A review of the challenges and proposals for improving patient access to advanced therapeutic medicinal products in the Spain

Executive Summary

RARE IMPACT is a multi-stakeholder initiative working to improve patient access to advanced therapy medicinal products (ATMPs). This patient-focused initiative aims to assess challenges and propose actionable solutions to concerns regarding access to these transformative rare disease treatments in Europe. Through engagement with health technology assessment (HTA) agencies, regulatory bodies, payers, patient groups, clinicians, manufacturers and other experts across Europe, RARE IMPACT partners identified challenges and have proposed solutions for better access to ATMPs in Europe.

This report aims to stimulate multi-level stakeholder discussions on patient access to ATMPs and is not intended to capture all challenges to patient access to ATMPs. The RARE IMPACT initiative was launched at the European Conference on Rare Diseases and Orphan Products in 2018.

A focus on cost-containment and affordability and the de-centralised nature of the pricing & reimbursement system has historically resulted in delays in patient access in Spain when compared with other EU5 countries

However, Spain has been actively addressing its approach to ATMPs and the National Strategy for Advanced Therapies has been put in place to improve access to these therapies. This has resulted in Kymriah and Yescarta being reimbursed much earlier than typically seen for orphan products in Spain.

P&R negotiations at the national and regional level have been focused on cost-containment and the near-term budget impact of the introduction of high-cost drugs. The assessment process in Spain is less rigorous than in other countries and there is no specific route for ATMPs. The opacity in assessment processes adds further to affordability challenges. Combined, these challenges make Spain a less attractive country for manufacturers to launch ATMPs, which is detrimental for patient access.

The model used to assess and grant reimbursement for CAR-Ts seeks to ensure equal access to high-cost medicines in all autonomous regions

The model requires monitoring of therapeutic effectiveness through a centralised procedure. The monitoring allows further information on the outcomes of the treatment to be collected to inform reimbursement discussions. This model could be a template for ATMPs in the future. Alongside providing equitable access, the model seeks to provide reimbursement at a national level for ATMPs. Innovative contractual agreements are not common in Spain, but they have been used for an ATMP in the past (ChondroCelect – payment-by-performance) and the finance models used for Yescarta and Kymriah also point to a willingness to engage in the development of innovative models.

A national level solution is important for ATMPs as otherwise, autonomous regions will conduct HTA processes and these can put greater emphasis on cost-effectiveness than those at the national level. It is likely that regional HTA will pose a major barrier to ATMP access given challenges associated with surrogate endpoints, outcomes, indirect treatment comparisons and evidence quality in rare diseases. Challenges concerning HTA requirements for ATMPs are common to orphan drug assessment generally. Small patient numbers in trials and limited duration of evaluation limits the efficacy and safety data that can be generated at the time of launch.

A focus on cost-containment in Spain could lead to use of hospital exemptions as a means to avoid reimbursement of commercially available products. The hospital exemption may pose a challenge to future patient access as it currently stands (although this issue tends to apply more to cell therapies), where products approved through the central authorisation process of the European Medicines Agency (EMA) may have to compete with products developed under a hospital exemption. This has implications for future product development and patient access to current and future products and undermines the stringent regulatory protocols products with Marketing Authorisation are required to undergo. To address this, the EU could issue guidelines defining more specifically the scope and requirements for hospital exemptions for ATMPs, stating clearly that when patients have access to an ATMP with a Marketing Authorisation, countries should not authorise hospital exemptions for the same medical indication.

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