A new position paper from the European University Hospitals Alliance sets out a road map that outlines the regulatory and organisational changes needed to bring ATMPs to more patients across Europe. Advanced therapy medicinal products (ATMPs) are at the very centre of healthcare innovation and are responsible for a paradigm shift in the treatment of many diseases. European research institutions contribute significantly to the development of such therapies, yet the number of cell and gene therapies that are introduced to the market remains limited. Current EU regulatory frameworks are not fully adapted to the specificities of these new therapies, thereby challenging optimal patient access and sustainability of these innovative products within European healthcare systems. EUHA is well positioned to make claims on ATMP development, as it brings together leading European academic medical institutions that have translated decades of research into approved therapies. Together, these institutions span the full ATMP pipeline (from discovery and Good Manufacturing Practice (GMP) manufacturing to clinical translation and approval) across multiple therapeutic areas. — Silvia Martin Lluesma, Author of the Paper and Head of Advanced Therapies Program at Vall d’Hebron Institute of Oncology Collaboration within EUHA enables shared expertise, infrastructure, and regulatory alignment, strengthening engagement with industry, small and medium-sized enterprises, and policymakers to advance the European ATMP ecosystem.” — Silvia Martin Lluesma, Author of the Paper and Head of Advanced Therapies Program at Vall d’Hebron Institute of Oncology Academic centres have a crucial role in the development of many ATMPs. However, the relative lack of funding and the complex regulatory framework in Europe put us at a disadvantage compared to other regions. — Johan Van Eldere, Author of the Paper and EUHA Secretary-General ‹ › A new position paper published in The Lancet Regional Health – Europe by experts from the European University Hospitals Alliance (EUHA) examines current challenges and sets out a series of concrete recommendations. The paper argues that the current regulatory and financial framework, while designed to protect patient safety, is not adequately adapted to the specific characteristics of ATMPs — and that targeted reforms are needed to improve both innovation and equitable patient access in Europe. The European Context ATMPs are not like conventional drugs. Their manufacturing is often tailored and personalised, their mechanisms of action are novel, and their regulatory and financial requirements reflect this complexity. Europe’s academic institutions and university hospitals are the primary drivers of early ATMP development. University hospitals play a central role in first-in-human clinical trials, bringing both scientific expertise and clinical knowledge to bear. However, the paper notes that despite promising preclinical results, many academic ATMP candidates fail to progress to clinical trials, largely due to limited resources and the complexity of regulatory pathways. Late-stage development remains heavily dependent on large biotech and pharmaceutical companies, which tend to prioritise higher-incidence indications over the rare diseases that many ATMPs are designed to treat. Regulatory complexity is also increasingly cited by innovators as a factor when deciding where to develop their products, at a time when the global landscape for advanced therapies is becoming more competitive. What EUHA Is Calling For In the roadmap recently published in The Lancet Regional Health – Europe, EUHA identifies several areas where reform is both needed and achievable: Updated Pre-clinical Pathways Standard animal models are poor predictors of ATMP outcomes. Organoids, AI-assisted modelling, and other emerging alternatives need to be formally validated and incorporated into regulatory frameworks. The European Medicines Agency (EMA), working with the European Commission and national authorities, must define harmonised, ATMP-specific pre-clinical requirements. Decentralised, Standardised Manufacturing Centralised, pharmaceutical-scale manufacturing is not suited to ATMPs. EUHA calls for the establishment of regional academic manufacturing platforms, supported by clear regulatory and logistical frameworks adapted to the unique requirements of small-scale and decentralised production models. Additionally, cross-border standardisation of GMP guidelines across member states is crucial for enhancing product quality and patient access, and for fostering innovation. A Dedicated Non-profit Pathway to Market Authorisation (MA) The costs of advancing an ATMP to marketing authorisation are prohibitive for academic institutions and non-profit organisations. EUHA calls for a dedicated non-profit MA pathway — with reduced fees, enhanced procedural support, and public funding mechanisms that extend through clinical trials and regulatory procedures. This must be backed by targeted funding calls within Horizon Europe and EU4Health for activities at Technology Readiness Levels 4–8, bridging the gap between the lab and the patient. Harmonised and Accessible Hospital Exemption (HE) Frameworks For ATMPs that will never attract commercial interest — rare disease somatic cell therapies, certain tissue-engineered products — the Hospital Exemption scheme is often the only access route. However, there is no harmonisation of eligibility criteria, quality standards, or data requirements across Member States. EUHA supports the continued use of hospital exemption while calling for meaningful harmonisation across borders through shared registries, equivalent safety and efficacy standards, and the integration of HE data into future marketing authorisation submissions when commercial development is viable. The goal is not to undermine EMA’s role, but to ensure that patients in smaller Member States, or those suffering from conditions too rare to attract industry attention, are not left behind. Real World Data and Evidence Standard drug evaluation requires large, randomised trials, often impractical for ATMPs. For this reason, the use of real-world evidence is crucial for alternative methodologies such as adaptive, dynamic designs of clinical trials. Innovative, Harmonised Reimbursement Pricing decisions for ATMPs are made at Member State level, using different methodologies, leading to wildly unequal access across Europe. For ultra-rare diseases, some ATMPs are simply never made available in smaller or lower-income Member States. EUHA proposes EU-harmonised pricing, reimbursement, and procurement mechanisms — including value-based pricing models, managed entry agreements, and centralised European negotiation for certain products, modelled on the joint procurement approach used for COVID-19 vaccines. Looking Ahead Europe has the scientific expertise, clinical infrastructure, and collaborative networks needed to lead the next generation of advanced therapies — but leadership will depend on whether policy can keep pace with innovation. As the EUHA position paper makes clear, the challenge is no longer simply discovering new treatments, but creating the regulatory, manufacturing, and reimbursement conditions that allow them to reach patients in Europe equitably and sustainably. If Europe can modernise its frameworks for ATMP development while preserving its commitment to safety, and quality, it has a unique opportunity to build a system that not only accelerates innovation but ensures that life-changing therapies are accessible to all patients who need them, regardless of geography or market size.