Regulatory Oversight of Cell and Gene Therapy Products in Malaysia.

The National Pharmaceutical Regulatory Agency (NPRA) is the agency responsible for the registration of pharmaceutical, natural, and health supplement products and notification of cosmetic products that are marketed in Malaysia. The implementation of regulatory oversight of the different types of product was in a progressive manner, with the latest addition to be regulated being the cell and gene therapy products (CGTPs), beginning January 1, 2021. CGTP can be classified as low risk (that does not require registration) or high risk (that needs to be registered). Generally, the regulation of high-risk CGTP is similar to other biological products. This chapter describes the chronology of the CGTP framework, classification of CGTP, how CGTPs fit into the current registration pathways and registration procedure, dossier requirements, and what is the current status and future direction of CGTP in Malaysia.

An Evaluation of Malaysian Regulatory Process for New Active Substances Approved in 2017 Using the OpERA Methodology.

INTRODUCTION: The National Pharmaceutical Regulatory Agency (NPRA) embarked on a regulatory-strengthening program and is evaluating its processes. Optimising Efficiencies in Regulatory Agencies (OpERA) is a regulatory-strengthening program that provides benchmarking data that can define performance targets and focus performance improvement. The objective of this study was to use OpERA methodology to determine where time is spent in the NPRA approval process and to form a baseline to measure the performance improvements. METHODS: The OpERA tool was used to collect specific milestone data that identify time periods, review stages, and data points for new active substances and biosimilars approved by NPRA in 2017. RESULTS: In 2017, 25 new active substances and 1 biosimilar were approved by NPRA in a median of 515 days, representing both agency and applicant time. The median time between dossier receipt and the initiation of NPRA scientific assessment was 135 days, but there was a wide variation in queuing time. The median total assessment time was 279 days (agency and applicant timing). NPRA took a median of 166 days; applicants took a median of 131 days to respond to deficiency questions, with up to 6 cycles of review required for approval and 65% of applications requiring 4-5 cycles to provide satisfactory responses. CONCLUSIONS: As a result of these data, NPRA proposes three improvements: target start for scientific assessment 100 days after file acceptance, a maximum of 5 review cycles, and applicant response time limited to 6 months. These results will serve as a baseline for further assessment.

Correction to: An Evaluation of Malaysian Regulatory Process for New Active Substances Approved in 2017 Using the OpERA Methodology.

The authors have revised Figure 1. The revised figure is presented below.