TABLE 4.

Comparison between the gene therapy regulatory framework in United States (US), European Union (EU), and Japan.

	Malaysia	US	EU	Japan
Key regulatory structures	• Ministry of Health (MOH) • National Pharmaceutical Regulatory Agency (NPRA) • National Stem Cell Research and Ethics Sub-committee (NSCERT) • Medical Research and Ethics Committee (MREC) • Institutional Review Board (IRB) or Institutional Ethical Board (IEB)	• Food and Drug Administration (FDA) • Center for Biologics Evaluation and Research (CBER) • 351 Product • Office of Tissues and Advanced Therapies (OTAT)	• European Medicines Agency (EMA) • Committee for Medicinal Products for Human Use (CHMP) • Advanced Therapeutic Medicinal Products (ATMP) • Committee for Advanced Therapies (CAT)	• Pharmaceuticals and Medical Devices Agency (PMDA) • Center for Product Evaluation • Regenerative Medicine Product • Office of Cellular and Tissue-based Products
Name of product	Cell and Gene Therapy Products (CGTPs)	Gene Therapy Product	Gene Therapy Medicinal Product (GTMP)	Gene Therapy Product
Definition of gene therapy	• Contains an active substance which consists of a recombinant nucleic acid administered to human beings with a view to regulate, repair, replace, add or delete a genetic sequence. • Its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of gene expression of this sequence.	• Mediate effects by transcription and/or translation of transferred genetic material and/or by integrating into the host genome and that are administered as nucleic acids, viruses, or genetically engineered microorganisms. • The products may be used to modify cells in vivo or transferred to cells ex vivo before being administrated to the recipient.	• Contains an active substance which consists of a recombinant nucleic acid administered to human beings to regulate, repair, replace, add or delete a genetic sequence. • Its therapeutic, prophylactic, or diagnostic effect relates directly to the product of genetic expression of this sequence.	• Articles which are intended to be used in the treatment of disease in humans or animals, and are transgened to express in human or animal cells.
Some main guidelines	• Guidance Document and Guidelines for Registration of Cell and Gene Therapy Products (CGTPS) in Malaysia (2016) • Checklist for Research on Stem Cell and Cell-based Therapies (NSCERT 2009)	• Long Term Follow-up After Administration of Human Gene Therapy Products; Guidance for Industry (2020) • Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products (2013) • Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy (1998)	• Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials (2019) • Quality, preclinical and clinical aspects of gene therapy medicinal products (2018) • Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells (2012)	• Regenerative Medicine Promotion Law (2013) • Act of Safety of Regenerative Medicine (2013) • Act on Pharmaceuticals and Medical Devices (2013)