Cell and gene therapy products in Malaysia: a snapshot of the industry's current regulation preparedness

Cell and gene therapy products (CGTPs) are anticipated to bring many benefits for the treatment of conditions with limited or no satisfactory treatment options. However, they are associated with concerns of potential safety risks because of their high complexity. The National Pharmaceutical Regulatory Agency (NPRA) of Malaysia took the first step toward the regulation of CGTPs by publishing the Malaysian Guidance Document and Guidelines for CGTPs in 2016. As mandatory registration and enforcement of CGTPs were scheduled to begin January 1, 2021, the aim of this study was to ascertain the industry's readiness for the regulation of CGTPs in terms of awareness of the guidelines, challenges and acceptance of the regulatory requirements. The authors invited 48 CGTP companies to participate in the survey between October 2019 and June 2020, and 30 companies responded. The majority of respondents were aware of the mandatory CGTP regulatory control and the availability of the guidelines. Many CGTPs were in the early development phase, and the most difficult registration barriers were dossier preparation and compliance with the pre-clinical and clinical requirements. These findings represent the current CGTP landscape in Malaysia from the industry's viewpoint, enabling the NPRA to implement initiatives to facilitate registration and enforcement.