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.

Mobile Technology in Medicine: Development and Validation of an Adapted System Usability Scale (SUS) Questionnaire and Modified Technology Acceptance Model (TAM) to Evaluate User Experience and Acceptability of a Mobile Application in MRI Safety Screening.

Background Magnetic resonance imaging (MRI) safety screening is a crucial procedure for patient preparation before entering into MRI room. Many hospitals in Malaysia are still using the MRI safety checklist printed form. Besides, clinicians will not get a definite conclusion about whether the patient is contraindicated for MRI or not. Hence, we have created one mobile application named MagnetoSafe to overcome this issue. The application will provide an instant decision on whether the patient has no contraindication, relative contraindication, or absolute contraindicated for MRI. We need to check for acceptability and user experience for any newly created mobile application. Objective This study was designed to check the validity of the adapted Technology Acceptance Model (TAM) and System Usability Scale (SUS) Questionnaire. Method The validity and reliability of the questionnaire were investigated. Subsequently, 52 fully completed responses were collected. Results Face and content validity of the questionnaires are considered acceptable with only minor changes to Item 10 of SUS. The Cronbach's alpha for the SUS questionnaire (10 questions) is -0.49, which is not acceptable. The Cronbach's alpha for TAM questionnaire (3 domains; 14 questions) is acceptable, which is 0.910 for perceived usefulness, 0.843 for perceived ease of use, and 0.915 for intention to use. Conclusion Face validity of the adapted SUS and modified TAM questionnaires is acceptable with only minor changes to Item 10 in SUS. Content validity with experts is good. However, the reliability of the SUS questionnaire is not acceptable and therefore adapted SUS will not be used for assessing user experience. The reliability of the modified TAM questionnaire with the original three-factor structure is considered acceptable and can be used to evaluate the user's acceptability of MagnetoSafe.

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

BACKGROUND AIMS: 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. METHODS: The authors invited 48 CGTP companies to participate in the survey between October 2019 and June 2020, and 30 companies responded. RESULTS: 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. CONCLUSIONS: 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.

An Update on the Registration of Biosimilars in Malaysia.

BACKGROUND: Because of its structure and complex manufacturing process, every biotherapeutic product (BTP; medicinal products made by or derived from living organisms and produced by biotechnology) adheres to stringent quality assurance and control requirements, in addition to extensive nonclinical and clinical study data. Similarly, copy products of original biotherapeutics (termed as "biosimilars") are subjected to equally strict regulatory control. BTPs have been registered in Malaysia since the 1990s; however, registration of biosimilars started only in 2008. METHODS: This research aims to compare evaluation practice on biosimilar and novel BTPs at the Biological Product Registration Section in Malaysia. Evaluation activities were studied in terms of evaluation questions, evaluation timeline, nonclinical and clinical requirements, and local requirements on product label (including package insert). Six biosimilar product dossiers and 6 novel BTP dossiers evaluated in 2013-2015 were sampled. Parameters for comparison were determined and analyzed using data collection forms. Specific to the biosimilar products, the evaluation practice on labels and package inserts were dissected and described in a qualitative arm of this research. RESULTS: Generally, the registration requirements of novel BTPs and biosimilar products are in agreement with international regulatory practices. However, some labeling and package insert requirements, and registration conditions are unique to the Malaysian regulatory context. CONCLUSIONS: Study findings revealed some similarities and differences in current evaluation practice (timeline and requirements) for biosimilar versus novel BTPs. The findings of this research also provide an insight on current evaluation practice on biosimilar product labeling.