One ATMP, Two Hospital Pharmacies, No Compromise

Our hospital pharmacy does not hold the regulatory authorization nor the technical infrastructure required to prepare Advanced Therapy Medicinal Products (ATMPs). However, we were urgently called to deliver gene therapy for a patient with recessive dystrophic epidermolysis bullosa, a rare and severely disabling genetic condition. This disease is caused by mutations in the COL7A1 gene, leading to a deficiency in type VII collagen and resulting in extreme skin fragility.
Beremagene geperpavec (B-vec), a gene therapy based on a modified herpes simplex virus type 1 (HSV-1) vector, became the first curative treatment targeting this disorder. It has been available in France through the early access program since September 2024.
The objective of this work was to develop a secure, regulatory-compliant pathway to ensure the weekly administration of B-vec within our facility, despite the absence of on-site preparation authorization. To achieve this, we established a partnership with a distant authorized hospital pharmacy located 150 km away.

Methods
We conducted a thorough review of regulatory requirements and scientific literature. An authorized pharmacy was identified for the sterile preparation of B-vec. A formal amendment to our existing service agreement was established to permit inter-institutional collaboration. The treatment was approved by our institution’s drug and therapeutics committee, despite its significant cost (>€24,000 per week).
We implemented a shared logistical workflow: weekly preparation by the authorized pharmacy, secure cold-chain transport, and controlled reception and dispensation at our hospital. Risk management protocols were validated by our infection control team. Nursing staff were trained using placebo preparations, and the first administration was supervised.

Results
The process enabled a high level of traceability, quality control, and patient safety. Collaboration between the dermatology department and the day care unit ensured clinical coordination. Hospital pharmacy technicians played a central role in the documentation, reception, and dispensing of the product.
To date, 23 weekly administrations have been performed without any incidents. The patient experienced significant improvements in comfort and quality of life.

Conclusion
This experience illustrates the practical, regulatory, and economic challenges posed by introducing ATMPs into non-accredited hospitals. While the inter-hospital collaboration model ensured timely and safe patient access to an advanced therapy, it remains fragile and heavily dependent on the resources of the authorized preparation center.
Looking ahead, the increasing use of ATMPs raises the need to reconsider national authorization models—either by expanding local preparation capabilities or creating regional centers. Above all, it calls for reflection on how to ensure equitable access to therapeutic innovation across all healthcare settings.

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