J Transl Med. 2026 May 23. doi: 10.1186/s12967-026-08303-x. Online ahead of print.
ABSTRACT
BACKGROUND: Advanced therapy medicinal products (ATMPs) comprise gene therapies, somatic cell therapies, tissue engineered products (TEPs), and combined ATMPs. These modalities have the potential to deliver durable or curative benefit in oncological, genetic, chronic, and rare diseases with high unmet medical need. Yet in Europe, access remains limited, uneven, and often commercially unsustainable: clinically promising therapies frequently fail to progress beyond early development or are withdrawn after regulatory approval for reasons related to scalability and reimbursement rather than lack of clinical benefit.
METHODS: This study was designed as a qualitative, interview-based analysis to identify the major barriers faced by ATMP stakeholders to the clinical translation and patient access of cell therapies in Europe. We collected structured oral and written questionnaire/interview input from six stakeholders operating in the European ATMP landscape and synthesized the material into cross-cutting themes related to health technology assessment, manufacturing, regulatory implementation, and market dynamics.
RESULTS: Stakeholders consistently report that HTA approaches optimized for conventional pharmacological therapies often overemphasize short-term budget impact and insufficiently account for long-term clinical benefit, downstream healthcare and societal costs, and the durability of therapeutic effects. In parallel, substantial variability across donors, batches, manufacturing and delivery settings, together with fragmented GMP infrastructure, uneven national execution of clinical trial and access pathways, and chronic underinvestment, undermines reproducibility and scalability. Although the European Medicines Agency (EMA) offers facilitation instruments including early scientific dialogue and accelerated or supportive regulatory pathways, their potential to improve predictability is frequently attenuated by Member State heterogeneity and discontinuities across regional innovation ecosystems.
CONCLUSION: From a stakeholder's perspective, key recommendations including revising HTA frameworks to better reflect long-term lifetime value, investing to improve the accessibility to GMP facilities, strengthening ATMP regulatory expertise and access to early scientific advice, enabling cross-border ecosystems, and addressing structural market failure through public risk-sharing mechanisms.
PMID:42177536 | DOI:10.1186/s12967-026-08303-x