The EBMT and GoCART brought together the key stakeholders across the European healthcare ecosystem — including research centres, healthcare professionals’ associations, and patient organisations at both EU and Member State levels to respond to the latest proposed changes to the Pharmaceutical regulation review currently in progress.
The key objective for all stakeholders is to preserve the original purpose and unrestricted scope of the Hospital Exemption (HE) for Advanced Therapy Medicinal Products (ATMPs) within the revised pharmaceutical framework. Since the adoption of the ATMP Regulation in 2007, the Hospital Exemption pathway has played a vital role in enabling timely, non-profit responses to unmet medical needs through the innovation of academic and hospital-based researchers. Evidence from the recent HaDEA Study confirms that HE-ATMPs represent the most personalised, affordable, and equitable form of innovative therapy currently available.
To ensure that Europe continues to lead in equitable therapeutic innovation, the EBMT and the co-signatories of this position statement want to urge EU legislators to refine the proposed pharmaceutical legislation so that it remains pragmatic, uniform, and ethically sound. The Hospital Exemption framework must continue to empower non-profit innovators to respond swiftly to patients’ unmet needs while upholding the highest standards of quality and safety.
In this spirit, the position statement puts forward the key recommendations to secure a fair, balanced, and effective regulatory environment for HE-ATMPs — one that sustains innovation, protects patient access, and strengthens Europe’s capacity to deliver advanced therapies for all. We remain committed to work constructively with EU institutions and agencies. Together, we can strengthen Europe’s leadership in Advanced Therapy Medicinal Products by fostering a regulatory environment that promotes innovation, safeguards patients, and supports equitable access to transformative therapies.