Particulates in CGT guidance survey results – an ISCT process development & manufacturing committee perspective on cell and gene therapy industry knowledge awareness

Abstract

The International Society for Cell & Gene Therapy Particulates Working Group has surveyed the global industry on commonly used regulatory guidance documents pertaining to particulates across various cell and gene therapy (CGT) product-based chemistry and manufacturing controls. Responses were collated from academic and industrially based individuals involved in or making decisions for manufacturing, quality control and analytics. Two surveys covering therapy developers and manufacturing suppliers were created and distributed via e-mail, through focus groups and via social media platforms. In total, 97 responses were received from CGT professionals at different career levels, which varied from technicians to corporate executives. The responses represent a snapshot of the different CGT industries, with a bias in cell therapy developers, because of the distribution of respondents. Overall, we observed a widespread use of common guidance and legislative documents with specific testing requirements pinned to certain CGT product types. Cell-based combination drugs and devices are not well addressed and may require other guidance that was not referenced. These results indicate that CGT particulate risk management should be clarified by collating various particulate guidelines into an easy-to-use guide that brings awareness to the various legislative and guidance documents pertaining to new and novel therapies, their classification, their route of administration and the regulatory jurisdiction under which they are handled. The group also recommends clearly outlining the differences and similarities in particulate management regulations across the globe. As the CGT industry is undergoing maturation, the risk analysis methods are also maturing. With such a broad range in product types, routes of administration, source materials and processing methods, standards of risk analysis and control need to be specific to the risk profile associated with any given drug product. The manufacturing strategy of novel drug types represented under the advanced therapy umbrella will require careful interpretation and implementation of the existing guidance identified in this survey, as not one guidance covers all manufacturing challenges with particulates.