[Translated article] New challenges in advanced therapies.

Area of Science:

• Pharmacology and Pharmaceutical Sciences
• Cellular and Molecular Medicine
• Hematology

Background:

• Advanced therapy medicinal products (AMTPs) are rapidly developing, posing significant challenges for hospital pharmacy operations.
• Hospital pharmacies must adapt to the complex logistical and regulatory demands of preparing AMTPs for both commercial and research purposes.

Purpose of the Study:

• To describe the implementation of an Advanced Therapies Unit (AUT) within a tertiary hospital.
• To detail the process of preparing AMTPs adhering to Good Manufacturing Practices (GMP).
• To present the outcomes of AMTP production, focusing on academic production challenges.

Main Methods:

• Establishment of an AUT compliant with GMP standards to ensure product quality and safety.
• Formation of a multidisciplinary team trained in GMP for the preparation of specific AMTPs, including allogeneic virus-specific T cells (VSTs).
• Development of a collaborative circuit involving the AUT, pharmacy, and hematology services for VST clinical application and preparation.

Main Results:

• The AUT successfully meets GMP requirements, guaranteeing the quality of prepared medicines.
• The established circuit facilitates the treatment of hematopoietic stem cell transplant patients with resistant viral reactivations using VSTs.
• Preliminary data indicate that VSTs are an effective and safe therapeutic option for specific patient populations.

Conclusions:

• Academic AMTPs, particularly for orphan indications, can benefit from hospital production via 'hospital exemption' for earlier, cost-effective access.
• Training hospital pharmacists in GMP is crucial for developing AMTPs that meet logistical and regulatory standards.
• The AUT model provides a framework for hospital pharmacies to manage the complexities of advanced therapy medicinal product preparation and support research.