Cell, Tissue and Gene Therapy Products
Cell, tissue, and gene therapy products represent a highly diverse range of medicinal products which continues to expand as the technology develops and matures. Their use in the treatment of human diseases and physical conditions has captured wide interest due to their potential to address many unmet medical needs. Examples include haematopoietic stem cells, skin grafts, expanded autologous cells, recombinant oncolytic viruses, novel gene editing technologies and engineered tissues. Their effectiveness has been demonstrated in the treatment of various conditions and illnesses such as haematological malignancies, burn treatment, thalassaemia, sickle cell disease, spinal muscular atrophy, certain cancers, and some monogenic inherited disorders.
With the growing diversity of types, and indications for the use, of cell, tissue and gene therapy products their regulatory oversight becomes increasingly crucial in order to ensure the accessibility to products with demonstrated quality, safety and efficacy, and which were obtained under acceptable ethical principles. Therefore, it is important to understand the products, their origins, manufacturing processes, mechanisms of action, and also to have appropriate classification schemes in place for their effective regulation. At the same time, it is important that regulatory environments do not hinder either their development or accessibility and so both regulatory harmonization and the use of regulatory reliance are strongly encouraged by WHO.
Cell, Tissue and Gene Therapy Product standardization
Written standards
At their March 2023 meeting, the WHO Expert Committee on Biological Standardization adopted their first guidance document specifically for cell, tissue and gene therapy products. This foundational document provides a set of definitions related to these products as well as a means for their classification. The document also encourages all countries establish a regulatory framework for such products, and to implement the concepts of regulatory cooperation, harmonization and reliance. Effective regulatory frameworks should help to improve access to these medicinal products while ensuring their continued quality, safety and efficacy.