The complex price setting and reimbursement of non-industrial advanced therapy medicinal products

Advanced Therapy Medicinal Products (ATMPs) developed under the Hospital Exemption (HE) offer innovative treatments for patients with unmet medical needs, but their pricing and reimbursement (P&R) remain fragmented and poorly understood. This paper examines the P&R landscape for HE-ATMPs developed by academic developers in Spain, based on the Spanish costing guideline and extends the analysis to HE-ATMPs developed by academic and non-profit developers across Europe. We conducted a mixed-method, exploratory study combining literature review with interviews and email enquiries of experts from five European countries to address gaps in evidence on P&R of ATMPs under the HE. We found that Spain is the only Member State to publish a detailed framework for calculating the price of academic ATMPs, incorporating direct production costs and a modest tiered research incentive. By contrast, other countries rely on Diagnosis Related Groups, ad-hoc negotiations, or lack formal reimbursement pathways altogether. Key challenges identified include methodological inconsistencies, limited transparency on costing methodology, absence of audit mechanisms, and debates around the inclusion of publicly funded R&D and long-term follow-up costs. Our findings highlight the need for harmonized, transparent, and standardized European costing methodologies to ensure fair pricing, sustainability of innovation, and equitable patient access to non-industrial ATMPs.