Abstract

The regulation of cell and gene therapy products (CGTPs) is essential for ensuring their safety, efficacy, and timely access to the market. This article describes Malaysia's current regulatory framework for CGTPs, emphasizing available pathways such as priority review, conditional registration, facilitated registration, and orphan drug designation. Additionally, this study employs a comparative analysis to examine expedited regulatory pathways and orphan drug designations in the United States (US), European Union (EU) and Japan, highlighting key differences that impact CGTP approvals. Notably, Malaysia utilises a broader regulatory approach, similar to EU’s PRIority Medicines (PRIME), in contrast to US and Japan which have specific pathways i.e. Regenerative Medicine Advanced Therapy (RMAT) and conditional and time limited designation, respectively. Facilitated registration pathway introduced in Malaysia is unique among the reviewed jurisdictions. It relies on foreign regulatory assessments and subsequently accelerating market access in Malaysia. The study concludes that existing regulatory pathways in Malaysia can be utilized by product registration holders to expedite CGTP approvals. Furthermore, adopting a tailored regulatory framework similar to those in other regions could enhance Malaysia's ability to support the development and commercialization of CGTPs, ultimately improving both patient safety and access to these innovative treatments.

Key words: Cell and Gene Therapy, Regulatory Pathways, Malaysia, Expedited Approval, International Comparison