Europe Maps Wider Access to Advanced Therapies

Advanced therapy medicinal products are opening treatment options for serious and rare diseases, including conditions previously without effective treatments. A 2026 Health Policy publication in The Lancet Regional Health – Europe sets out a European roadmap for regulatory, organisational and financial reforms to support their development and clinical use. These therapies differ from conventional drugs and tissues through their manufacturing, quality control, mechanisms of action and frequent single-use administration. Their implementation in European health systems remains constrained by regulation, high costs, production capacity limits and uncertainty around long-term outcomes. The roadmap places particular emphasis on academic organisations, university hospitals and small and medium-sized enterprises, which drive much early development but often struggle to move promising candidates into clinical trials and wider access. It also frames access as a regional, national and European responsibility.

Must Read: WHO Progress Falls Short of Global Health Targets

Regulatory Pathways and Manufacturing Capacity

Current European frameworks do not fully match the characteristics of advanced therapy medicinal products. Conventional pre-clinical testing can be difficult to apply because animal models often offer limited prediction of clinical outcomes. Alternative approaches, including organoids, three-dimensional cell clusters and artificial intelligence modelling, require further development and regulatory incorporation. Gene editing technologies also create specific safety questions, including off-target effects, malignant transformation risks and immunogenicity. Clearer harmonised requirements from European bodies could help guide progression towards first-in-human clinical testing while keeping safety standards central. The 2027 regulation for products derived from substances of human origin creates a non-profit framework that could support faster and more equitable access for some minimally manipulated somatic cell and tissue-based therapies.

Manufacturing creates another major barrier. Many development activities take place in academic institutions or small and medium-sized enterprises but transfer to larger organisations capable of safe and cost-effective marketing can be problematic. Standardised and decentralised production could improve access, particularly through academic centres and disease-specific networks. A hub-and-spoke model may suit larger indications, while autologous cell therapies may benefit from point-of-care production because they require intensive personalised interaction with healthcare professionals. Regional academic manufacturing platforms, combined with aligned rules for testing, validation, raw materials, quality control, outcome measurement and long-term follow-up, could support consistent product quality and cross-border development.

Clinical Development and Evidence Needs

Academic and non-profit advanced therapy developers face major bottlenecks in clinical development. Regulatory complexity and limited funding make progression towards marketing authorisation difficult, especially because manufacturing, clinical trials and regulatory standards involve high costs. Early-phase trials may be possible in academic centres, but late-stage development often depends on large biotech and pharmaceutical companies. This creates difficulties for rare diseases, where commercial incentives may be weaker than for high-incidence indications. Public funding schemes often focus on basic research and early innovation, leaving limited support for clinical trials, safety studies and regulatory procedures.

A dedicated non-profit route to marketing authorisation could improve patient access. Such a pathway would include reduced or waived regulatory fees, procedural support and targeted public funding for activities between pre-clinical work and late-stage trials. Stronger collaboration between academic developers, industry partners and regulatory agencies could also support translation from laboratory work to clinical use. Hospital exemption remains relevant for products without commercial viability. Harmonised eligibility criteria, quality standards and data requirements across Europe could protect safety and credibility without undermining central European medicines evaluation. Shared registries, cross-border access mechanisms and integration of hospital exemption data into later regulatory submissions could help small patient populations when full authorisation is not feasible.

Adaptive Evidence and Sustainable Payment

Standard drug evaluation based on large randomised trials is often difficult for advanced therapy medicinal products, particularly in rare diseases, precision medicine and frail populations. Single-arm trials, external comparators, adaptive designs and real-world data can support development when conventional trial structures are not practical. These methods require rigorous statistical planning to avoid bias, multiple testing problems and misleading post-hoc changes. Real-world data use remains complicated by privacy requirements, ethics approvals and fragmented product-based registries, while registry duplication increases the burden on clinicians. A federated European infrastructure could support pharmacovigilance and meet both regulatory and payer requirements. Specialist centres also need the capability to monitor patients closely, provide rapid treatment options and coordinate long-term follow-up.

Financial arrangements also limit widespread clinical use. Advanced therapy medicinal products may deliver major health benefits, but short-term budget effects can still prevent payment. Many reach the market before long-term safety and efficacy data are available, while delaying authorisation to wait for such data would slow patient access. European marketing authorisation is centralised, while health economic assessment and pricing remain with individual Member States. Innovative reimbursement options include value-based pricing, managed entry agreements, dynamic pricing, annuity payments and outcome-based models. Joint procurement and voluntary cross-border agreements could help address market fragmentation, especially for ultra-rare therapies that may not become available in every country.

Advanced therapy medicinal products require regulatory, clinical and financial approaches that reflect their distinctive manufacturing, evidence and access challenges. Europe needs proportionate safeguards that protect patients without blocking innovation, especially for rare and commercially non-viable conditions. Academic organisations, university hospitals and small and medium-sized enterprises need clearer routes through clinical development, market access and reimbursement. Harmonised hospital exemption, adaptive evidence pathways, decentralised manufacturing and sustainable payment models could help turn promising therapies into accessible treatments across European health systems while preserving safety, quality and long-term evidence generation.

Source: The Lancet Regional Health – Europe

Image Credit: iStock