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Regulatory Oversight of Cell and Gene Therapy Products in Canada.
Adv Exp Med Biol. 2015; 871:49-71.AE

Abstract

Health Canada regulates gene therapy products and many cell therapy products as biological drugs under the Canadian Food and Drugs Act and its attendant regulations. Cellular products that meet certain criteria, including minimal manipulation and homologous use, may be subjected to a standards-based approach under the Safety of Human Cells, Tissues and Organs for Transplantation Regulations. The manufacture and clinical testing of cell and gene therapy products (CGTPs) presents many challenges beyond those for protein biologics. Cells cannot be subjected to pathogen removal or inactivation procedures and must frequently be administered shortly after final formulation. Viral vector design and manufacturing control are critically important to overall product quality and linked to safety and efficacy in patients through concerns such as replication competence, vector integration, and vector shedding. In addition, for many CGTPs, the value of nonclinical studies is largely limited to providing proof of concept, and the first meaningful data relating to appropriate dosing, safety parameters, and validity of surrogate or true determinants of efficacy must come from carefully designed clinical trials in patients. Addressing these numerous challenges requires application of various risk mitigation strategies and meeting regulatory expectations specifically adapted to the product types. Regulatory cooperation and harmonisation at an international level are essential for progress in the development and commercialisation of these products. However, particularly in the area of cell therapy, new regulatory paradigms may be needed to harness the benefits of clinical progress in situations where the resources and motivation to pursue a typical drug product approval pathway may be lacking.

Authors+Show Affiliations

Centre for Evaluation of the Radiopharmaceuticals and Biotherapeutics, Biologics and Genetic Therapies Directorate, Health Products and Food Branch, Health Canada, 100 Eglantine Driveway, A/L 0603C, Ottawa, ON, Canada, K1A0K9. anthony.ridgway@hc-sc.gc.ca.Centre for Biologics Evaluation, Biologics and Genetic Therapies Directorate, Health Products and Food Branch, Health Canada, 100 Eglantine Driveway, A/L 0603C, Ottawa, ON, Canada, K1A0K9.Clinical Evaluation Division - Haematology/Oncology, Centre for Evaluation of Radiopharmaceuticals and Biotherapeutics, Biologics and Genetic Therapies Directorate, Health Products and Food Branch, Health Canada, 100 Eglantine Driveway, A/L 0603C, Ottawa, ON, Canada, K1A0K9.Stem Cell Research Lab, Centre for Biologics Evaluation, Biologics and Genetic Therapies Directorate, Health Products and Food Branch, Health Canada, 251 Sir Frederick Banting Driveway, A/L 2201E, Ottawa, ON, Canada, K1A0K9.

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

26374212

Citation

Ridgway, Anthony, et al. "Regulatory Oversight of Cell and Gene Therapy Products in Canada." Advances in Experimental Medicine and Biology, vol. 871, 2015, pp. 49-71.
Ridgway A, Agbanyo F, Wang J, et al. Regulatory Oversight of Cell and Gene Therapy Products in Canada. Adv Exp Med Biol. 2015;871:49-71.
Ridgway, A., Agbanyo, F., Wang, J., & Rosu-Myles, M. (2015). Regulatory Oversight of Cell and Gene Therapy Products in Canada. Advances in Experimental Medicine and Biology, 871, 49-71. https://doi.org/10.1007/978-3-319-18618-4_3
Ridgway A, et al. Regulatory Oversight of Cell and Gene Therapy Products in Canada. Adv Exp Med Biol. 2015;871:49-71. PubMed PMID: 26374212.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Regulatory Oversight of Cell and Gene Therapy Products in Canada. AU - Ridgway,Anthony, AU - Agbanyo,Francisca, AU - Wang,Jian, AU - Rosu-Myles,Michael, PY - 2015/9/17/entrez PY - 2015/9/17/pubmed PY - 2015/12/30/medline KW - CTO Regulations KW - Health Canada KW - More than minimally manipulated KW - Regulation of cell therapy KW - Regulation of gene therapy KW - Safety of Human Cells, Tissues and Organs for Transplantation Regulations SP - 49 EP - 71 JF - Advances in experimental medicine and biology JO - Adv. Exp. Med. Biol. VL - 871 N2 - Health Canada regulates gene therapy products and many cell therapy products as biological drugs under the Canadian Food and Drugs Act and its attendant regulations. Cellular products that meet certain criteria, including minimal manipulation and homologous use, may be subjected to a standards-based approach under the Safety of Human Cells, Tissues and Organs for Transplantation Regulations. The manufacture and clinical testing of cell and gene therapy products (CGTPs) presents many challenges beyond those for protein biologics. Cells cannot be subjected to pathogen removal or inactivation procedures and must frequently be administered shortly after final formulation. Viral vector design and manufacturing control are critically important to overall product quality and linked to safety and efficacy in patients through concerns such as replication competence, vector integration, and vector shedding. In addition, for many CGTPs, the value of nonclinical studies is largely limited to providing proof of concept, and the first meaningful data relating to appropriate dosing, safety parameters, and validity of surrogate or true determinants of efficacy must come from carefully designed clinical trials in patients. Addressing these numerous challenges requires application of various risk mitigation strategies and meeting regulatory expectations specifically adapted to the product types. Regulatory cooperation and harmonisation at an international level are essential for progress in the development and commercialisation of these products. However, particularly in the area of cell therapy, new regulatory paradigms may be needed to harness the benefits of clinical progress in situations where the resources and motivation to pursue a typical drug product approval pathway may be lacking. SN - 0065-2598 UR - https://www.unboundmedicine.com/medline/citation/26374212/Regulatory_Oversight_of_Cell_and_Gene_Therapy_Products_in_Canada_ L2 - https://dx.doi.org/10.1007/978-3-319-18618-4_3 DB - PRIME DP - Unbound Medicine ER -