Despite regulatory success in Europe and the UK, access to CAR-T therapies remains inconsistent and often delayed. In his latest commentary, Dr João Gonçalves PhD examines how misalignment between approval, reimbursement and hospital implementation continues to restrict patient access and asserts that hospital pharmacists have a pivotal role in bridging this gap.
Six CAR-T products have been granted marketing authorisation by the European Medicines Agency (EMA) across the European Economic Area, collectively covering 15 haematologic malignancies – a notable regulatory achievement that reflects years of scientific rigour and a strong commitment to advancing transformative therapies.1
However, the current state of CAR-T therapies presents a paradox, as many patients across member states and hospital settings still cannot access these treatments.
This issue stems not only from a lack of approval or limited evidence, but also from systemic failures in the process from regulatory approval to actual patient access – problems that the EMA alone cannot resolve.
In Europe, CAR-T therapies benefit from expedited regulatory pathways designed to accelerate market access and foster innovation to meet unmet medical needs. Yet after approval and European Commission endorsement, progress frequently stalls, leaving eligible patients without timely access.
Where CAR-T access breaks down
The roots of this paradox are well documented. Health technology assessment (HTA) processes were developed in an era focused on chronic disease management, incremental pharmacological innovation and reliance on extensive long-term randomised controlled trials.
By contrast, CAR-T therapies are supported by single-arm studies in rare indications. They have limited long-term follow-up, complex patient-specific manufacturing and high upfront costs that do not align with diagnosis-related, group-based hospital funding models.
With costs often exceeding €350,000 per patient per dose, disparities in access constitute a bureaucratic challenge and a significant issue of clinical equity. For patients with relapsed or refractory haematologic malignancies, whose survival may be measured in months, delays in access have direct consequences on outcomes.
While payer caution is understandable, the tools currently used – including cost-per-quality-adjusted life year analyses, budget-impact models and evidence certainty frameworks – were not designed for advanced therapy medicinal products (ATMPs) such as CAR-T therapies, thereby contributing to systematic delays in decision-making.
HTA and CAR-T reimbursement
National HTA bodies independently assess clinical and economic value for therapies, each using different methodological frameworks, cost-effectiveness thresholds and budget-impact criteria.
The result is a fragmented landscape: the same product may be reimbursed and integrated into clinical practice in one country, restricted to a narrow patient subset in another, pending assessment for years in a third and effectively unavailable in a fourth.2
An analysis of reimbursement decisions by HTA bodies across G7 countries showed significant heterogeneity. France and Germany recommended funding for 92% of CAR-T indications assessed, while England funded only 50% and Australia just 33%.3
Such variation illustrates how the same EMA-approved evidence base can yield markedly different coverage outcomes depending on the national HTA framework applied.
These differences translate into clear geographic inequities. Data from the European Society for Blood and Marrow Transplantation (EBMT) registry show over 10,000 CAR-T infusions across more than 600 centres in 60 countries as of September 2024, with activity concentrated largely in Western Europe. In contrast, several Eastern European nations have yet to report any commercial CAR-T use.4
A recent cross-country analysis showed that, despite EMA authorisation, CAR-T therapies had not been introduced in all European countries. For example, Latvia, Hungary and Malta offered only limited or no access to these treatments, and Norway was unable to provide certain approved products domestically.5
European policy responses
The European Union has recognised these disparities. Proposed reforms to pharmaceutical legislation aim to incentivise marketing authorisation holders to launch products across all member states, rather than limiting launches to the largest markets, by offering extended data protection periods.1
In parallel, the joint clinical assessment framework introduced under the EU HTA Regulation, effective for ATMPs from January 2025, aims to harmonise clinical evidence reviews, reduce duplication and establish a shared foundation to facilitate more efficient national pricing and reimbursement negotiations.6
Studies on stakeholder readiness for this reform emphasise the need for clearer communication on how these joint clinical assessments will influence national decision-making, alongside the development of measurement frameworks and ongoing stakeholder collaboration to ensure effective implementation.7
While these steps are significant, progress remains gradual and does not yet extend to the hospital level, where access is ultimately determined.
Outcomes-based agreements and real-world evidence
Outcomes-based managed entry agreements (OBMEAs), which have been adopted in several European countries to address the cost and evidentiary uncertainty of CAR-T therapies, present both opportunities and challenges for hospital pharmacy.8
OBMEAs can facilitate reimbursement for therapies that payers might otherwise decline to fund under conventional models. For example, Italy and Spain employ individual performance-based agreements that link payment to patient response, while Belgium, England and France utilise population-based agreements to enable access and generate the real-world evidence required for re-appraisal.8
However, OBMEAs require robust, auditable and sustainable data infrastructures at the hospital level, which must be established, maintained and governed – responsibilities that typically fall to hospital pharmacy within integrated care models.
Evidence highlights significant operational challenges in implementing OBMEAs for ATMPs, including the need for substantial upfront investment, well-defined governance structures for contracts and clearly delineated roles for clinical teams in data collection to minimise conflicts of interest.9
When properly resourced, these systems can enhance hospital bargaining power and boost resilience against disruptions in access. Initiatives such as the GoCART Coalition offer frameworks for standardising programme accreditation, quality assurance and evidence generation across European centres.
Hospital pharmacists facilitating CAR-T access
At this level, hospital pharmacists play a critical but often under-recognised role as facilitators of access. Traditionally associated with operational responsibilities, such as coordinating leukapheresis, cold-chain logistics, product release and toxicity management, their role increasingly encompasses strategic and governance domains.
Pharmacists in established CAR-T centres contribute to patient selection in line with reimbursement criteria, design governance frameworks that link clinical eligibility to budget approval, and manage the interface between EMA approval, hospital financing and patient care.
The European Association of Hospital Pharmacists (EAHP) also advocates that hospital pharmacists participate in HTA bodies at both national and regional levels and serve as advisory experts in central price negotiations.10
Indeed, HTA literacy – the ability to interpret and apply documents from decision-making bodies or joint clinical assessment reports – is now a core competency for hospital pharmacists involved in delivering CAR-T therapy. This reflects the profession’s unique position at the intersection of clinical, regulatory and economic systems, making it essential to access governance.
Additional essential skills include economic evaluation, management of risk-sharing agreements, governance of outcomes registries and effective communication with clinical teams, hospital administrators, payers and regulators.
Building reform and implementation
Despite these developments, the need for macro-level reform remains urgent. Joint HTA processes, legislative incentives for pan-European market launches, harmonised hospital exemption frameworks and expedited conditional reimbursement pathways are essential, but progress is often slow, contested and influenced by national health policy sovereignty.
In the meantime, the CAR-T access paradox remains a significant challenge. Eligible patients continue to seek treatment at centres developing programmes and negotiating reimbursement, where hospital pharmacists are already actively involved in these decision-making processes.
The key issue is whether the profession is adequately prepared and supported to fully take on these responsibilities and make these decisions. Hospital pharmacists already have many of the necessary tools to meet this challenge. What remains essential is ongoing commitment and focused investment to turn these capabilities into practice.
Author
João Gonçalves PharmD PhD
Faculty of Pharmacy, University of Lisbon and Imed Research Institute for Medicines, Lisbon, Portugal
References
1 Pennings ERA et al. The European CAR-T map – Current status and future directions to improve access to CAR T-cell therapy for hematologic malignancies. HemaSphere 2026;10:e70306.
2 Gonçalves E. CAR-T cell therapies: patient access and affordability solutions. Future Sci OA 2025;11(1):2483613.
3 Ge A et al. Global Access to Chimeric Antigen Receptor (CAR) T-Cell Therapies: Health Technology Assessment (HTA) of CAR T in G7 Countries and Australia. Blood 2024;144(Suppl 1):3659.
4 EBMT. The EBMT reaches major milestone: 10,000 CAR-T treated patients registered in its Registry. 9 September 2024. [Accessed April 2026].
5 Litvinova Y et al. Availability and financing of CAR-T cell therapies: A cross-country comparative analysis. Health Policy 2024;149:105153.
6 European Commission. New EU rules on Health Technology Assessment open up a new era for patient access to innovation. 2025;Jan 10.
7 Wang T, McAuslane N. Ensuring the Efficiency and Effectiveness of Joint Clinical Assessment in National HTA Decision-Making: Insights from the 2024 CIRS Multi-Stakeholder Workshop. J Mark Access Health Policy 2025;13(1):9.
8 Facey K et al. Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel. Pharmacoeconomics 2021;39(9):1021–44.
9 Greco A et al. A Systematic Review of Challenges and Opportunities in the Implementation of Managed Entry Agreements for Advanced Therapy Medicinal Products. Clin Ther 2025;47(2):e16–e26.
10 EAHP. Position Paper on Procurement. June 2024. [Accessed April 2026].
