New challenges of advanced therapies

Area of Science:

• Cellular therapy
• Pharmacology
• Hospital pharmacy

Background:

• Advanced therapy medicinal products (ATMPs) are rapidly developing, posing challenges for hospital pharmacy.
• Hospital pharmacies must adapt to the production and quality control of these complex therapies.
• Academic production of ATMPs requires specific infrastructure and expertise.

Purpose of the Study:

• To describe the implementation of an Advanced Therapies Unit (AUT).
• To detail the process of preparing ATMPs under "good manufacturing practices" (GMP).
• To present results from an academic ATMP production in a tertiary hospital.

Main Methods:

• Establishment of an AUT meeting GMP requirements.
• Formation of a multidisciplinary team trained in GMP.
• Development of a collaborative circuit for VST preparation and administration.

Main Results:

• The AUT ensures ATMP quality and supports research.
• The unit is authorized for five types of allogeneic virus-specific T cells (VSTs).
• A collaborative circuit enables VST treatment for viral reactivations post-transplant.

Conclusions:

• Preliminary results indicate VSTs are effective and safe.
• Hospital production via "hospital exemption" can improve early access to orphan ATMPs.
• Training hospital pharmacists in GMP is crucial for ATMP development and regulatory compliance.