CAR-T cell therapies have ushered in remarkable advancements in treating cancers such as lymphoma, leukemia, and multiple myeloma. By genetically engineering patients’ immune cells to combat their disease directly, these therapies offer unprecedented hope. However, the prohibitive costs of commercial CAR T-cell products, ranging from USD 350,000 to over USD 500,000 per patient, present an enormous barrier to widespread adoption and accessibility.
The University Medical Center Groningen (UMCG) in the Netherlands has taken steps toward addressing these issues by developing an affordable alternative to commercially available CAR-T cell therapies. This “in-house” treatment has undeniable advantages: faster production times (one to two weeks compared to four to six weeks for commercial options), improved quality due to the immediate processing of fresh cells, and notably lower calculated costs – around €80,000 (approximately USD 90,500) per patient. This initiative rightly evokes admiration and demonstrates the immense possibilities when public institutions prioritize patient welfare over market-driven imperatives.
The limits of the hospital exemption path
Yet, rather than fully seizing this opportunity to challenge a pharmaceutical industry dominated by profit-driven logic, UMCG is pursuing the so-called “hospital exemption.” Under this provision, hospitals can use therapies internally without requiring full marketing authorization from the European Medicines Agency (EMA). While praiseworthy in reducing short-term expenses, bypassing EMA regulatory pathways comes with significant drawbacks.
Firstly, by not obtaining EMA approval, UMCG’s therapies lack wider market access across Europe, depriving potential patients from benefits. Secondly, an EMA registration ensures rigorous quality control and continuous therapy safety and efficacy monitoring despite initial costs and complexities. The hospital exemption approach might inadvertently promote conditions with variable quality standards across different facilities, potentially risking patient safety.
A particularly important concern, voiced by academic developers, such as those behind ARI-0001 in Spain, is the lack of harmonization of hospital exemption frameworks across EU member states. While nine countries have implemented hospital exemption rules, discrepancies in regulatory requirements, monitoring procedures, and quality control standards pose challenges to both transparency and equity in patient access. This fragmented landscape may undermine confidence in the reliability and reproducibility of in-house CAR-T therapies across borders, even when such therapies demonstrate safety and efficacy comparable to commercial alternatives. Thirdly, commercial entities investing heavily in CAR-T therapies are unlikely to reduce prices without competitive pressure from approved and broadly accessible academic therapies. UMCG is missing the opportunity to reshape market dynamics more permanently and effectively by choosing exemption over full EMA approval.
Big Pharma tightens its grip on CAR-T
At the same time, major pharmaceutical companies are rapidly expanding their presence in the CAR-T cell therapy market, especially in Europe. AstraZeneca’s billion-dollar acquisitions of EsoBiotec and Gracell Biotechnologies claim to streamline production and reduce costs, yet ultimately consolidate corporate control over treatments that should ideally remain publicly accessible. Bristol Myers Squibb’s expansion in Leiden, Gilead’s Amsterdam facility, Novartis’s decentralized production initiatives, and Johnson & Johnson’s collaboration with Legend Biotech in establishing a CAR-T cmanufacturing facility in Ghent further underscore this troubling trend. While these developments may superficially improve treatment availability, the record consistently demonstrates that corporate-led initiatives prioritize profit margins and proprietary technology over sustainable affordability and equitable access.
Nevertheless, genuine alternatives exist. Public pharma initiatives, including Canada’s decentralized CAR-T production and India’s NexCAR19, demonstrate that advanced treatments can be delivered affordably, sometimes at less than one-tenth of the commercial cost, without compromising quality or safety.
In Europe, Amsterdam’s Antoni van Leeuwenhoek Hospital (AVL) offers a visionary example by actively seeking full EMA approval for its own cell therapies. AVL’s decision not only ensures rigorous quality and safety standards but also provides a scalable model that other hospitals across the region can adopt. However, it is also essential to acknowledge that AVL’s path to EMA approval is comparatively straightforward, as there are currently no EMA-approved commercial tumor-infiltrating lymphocyte (TIL) cell treatments. By contrast, UMCG faces a more complex regulatory environment, as several commercially-produced CAR-T cell therapies already hold EMA approval for similar indications, posing a more significant challenge for obtaining full regulatory approval.
No successful innovation without universal access
Despite these complexities, UMCG stands at a critical point in time. Choosing for the limited hospital exemption route only threatens to ingrain a healthcare model where equitable access remains an exception rather than the norm, inadvertently sustaining healthcare as a privilege rather than a universal right. This provision should not be viewed as an endpoint but rather as a strategic intermediate step. The Spanish experience with ARI-0001 has shown that hospital exemptions can effectively bring therapies to patients with urgent unmet needs while maintaining stringent quality and safety standards. Moreover, with continued data collection and regulatory dialogue, hospital exemption-based therapies can transition toward full EMA authorization – ultimately achieving broader access and sustainability.
Healthcare innovation must prioritize equitable, sustainable solutions accessible to all patients in need. By treating the hospital exemption not as a substitute but as a bridge to centralized EMA approval, UMCG can preserve its commitment to affordability and public service while also setting a powerful precedent. This path would uphold rigorous standards, inspire other academic centers, and reinforce the case for a harmonized, patient-focused regulatory framework that makes advanced therapies universally accessible – not only to those who can afford them, but to all who need them.
People’s Health Dispatch is a fortnightly bulletin published by the People’s Health Movement and Peoples Dispatch. For more articles and to subscribe to People’s Health Dispatch, click here.
David Franco is a scientist and public health activist based in Belgium. In the People’s Health Movement, he focuses on the Public Pharma for Europe (PPfE) initiative. David holds a PhD in Pharmaceutical Sciences from the Free University of Brussels.
