[Translated article] New challenges in advanced therapies

Abstract

The huge development that advanced therapy medicinal products (AMTPs) have experienced in recent years, both commercial and research, represent a challenge for hospital pharmacy at all levels. The aim of this article is to describe the implementation of an advanced therapies unit (AUT) and the process of preparation of the AMTPs according to the “good manufacturing practices” (GMP), as well as the results obtained in a tertiary hospital, as an example of the challenges posed by MTA's academic production.

The AUT meets the requirements established in the GMP by guaranteeing that the medicines produced therein are of the quality required for the use for which they are intended, and also provides support to various research groups involved in the development of AMTPs. The AUT is composed of a highly qualified multidisciplinary team, qualified and trained in GMP, and is authorized for the preparation of 5 types of AMTPs consisting of allogeneic virus-specific T cells (VST) with various viral specificities. A circuit has been established in collaboration between the UTA and the pharmacy service with the hematology service for the assessment of the clinical indication, the request, and preparation of VST, which allows the treatment of patients receiving hematopoietic stem cell transplants who present viral reactivations resistant or refractory to standard treatment, or who cannot tolerate it due to toxicity. Preliminary results from these AMTPs suggest that VSTs are an effective and safe alternative.

Academic AMTPs have special interest in orphan indications or in the absence of alternative treatments, and their production through the “hospital exemption” can favor early access in the initial phases of development and at a lower cost. It is essential to promote the training of hospital pharmacists in GMP and their participation in collaboration with other clinicians and researchers to develop AMTPs that meet all logistical and regulatory requirements.