Quality compliance in the development of cell-based medicines in non-pharma environments

Abstract

Background Academic institutions and transfusion centres are positioned to lead the development of novel cell-based therapeutics up to early-stage clinical trials (and further seeking marketing authorisation) or, alternatively, its use under the hospital exemption clause [1,2]. Existing structures, including blood and tissue bank facilities, translational medical programs and skilled personnel experienced in cell culture technologies and transplantation, equips these non-commercial entities to translate research-grade cell materials into safe and commercially viable products suitable for human use. However, developers must negotiate the complex regulatory and quality requirements involving the compulsory compliance of Good Scientific Practice (GxP) regulations in the development of advanced therapy medicinal products (ATMP) [3]. Originally designed for corporate environments, implementation of GxP in non-pharmaceutical institutions may consume a considerable level of effort (particularly in terms of funding and human resources). In non-pharmaceutical environments, current quality management structures for cell therapies are highly specific for blood and bone marrow-related products, including programs administered by JACIE, NetCord, FACT, AABB, or focused upon more global quality objectives, such as ISO guidelines (particularly ISO9001). The adaptation of such either general or specific quality assurance programs into GxP may appear as a substantial effort. However, many of the requirements are common across standards and the existence of any of them in a given institution can suit up to some extent to some of the aspects covered by the GxP. Rather than focusing on each certification separately, product developers and manufacturers in all environments would benefit from a single quality management system incorporating the most convenient tools available from different standards. In the present work, we analysed the GxP requirements and those in voluntary quality accreditation schemes already present in any blood and tissue bank. A number of common aspects were found, therefore offering an opportunity to avoid duplicities in the implementation process. Our work aims at serving as a guide for the optimisation of resources available and contributes to moving towards a single quality assurance system that integrates others, thus improving efficiency.