STANDARDS OF CARE

CONTEXT 

Gene and cellular therapies are inherently complex products and treatment administration is restricted to qualified centres. With the rapid developments and pending product approvals, there is a need for developing treatment guidelines and harmonising centre qualification procedures across pharmaceutical companies, accreditation bodies and national requirements.

OVERALL AIMS

1. Develop harmonised guidelines on patient and product management for health care professionals.

2. Reduce inspection burden and redundancies by developing and implementing consensus-driven requirements and qualification standards for clinical teams delivering gene and cellular therapies from cells and tissues of haematopoietic origin.

CURRENT WORK GROUPS/ACTIVITIES

Harmonise centre qualification strategies by leveraging the Joint Accreditation Committee, International Society of Cellular Therapy-Europe & EBMT (JACIE) qualification scheme

Harmonise labelling, documentation and packaging requirements

Develop guidelines on patient and product management, topics of interest include:
·    Guidance for hospital pharmacies on the implementation and operational management of CAR T.
·    Recommendation guidelines on apheresis for Advanced Therapy Medicinal Products (ATMPs) manufacture.

MAIN ACHIEVEMENTS TO DATE

We launched a committee to work on the harmonisation of existing centre qualification schemes to reduce inspection burden for centres. Through this successful interaction with stakeholders we were able to announce on 8 March 2022 a new initiative to allow apheresis and cell therapy treatment centres accredited by JACIE to leverage their accreditation as part of site qualification processes for Bristol Myers Squibb (BMS), Janssen, Kite and Novartis. Subject to marketing authorisation holders’ agreement and obligations, centres holding JACIE accreditation will have the option of undergoing a reduced or remote audit focused on product-specific requirements for cell therapies. The ongoing work with BMS, Janssen, Kite and Novartis includes specifying what this leverage means at a company level and working with other cell and gene therapy companies to join in leveraging the JACIE accreditation. 

In 2023, the GoCART Coalition Pharmacist Working Group published a practical guidance on implementation and safe operational use of marketed CAR T products within hospital pharmacies primarily throughout Europe. The guideline outlines the key areas where pharmaceutical expertise should focus on as well as key considerations for the hospital pharmacies. 

The GoCART Coalition Apheresis Working Group has worked on guidelines on ‘best practice’ in apheresis for optimal starting materials. These guidelines will be published in two manuscripts. The first manuscript, published in 2023, focuses on pragmatic aspects of apheresis in order to optimise the collection including venous access and apheresis platforms and technical aspects of the procedure. The second manuscript will be developed to describe washout phases and manufacturing failures; sampling of the apheresis product and labelling and cryopreservation as well as provide an outlook.