Chapter title |
Regulatory Oversight of Cell and Gene Therapy Products in Canada
|
---|---|
Chapter number | 3 |
Book title |
Regulatory Aspects of Gene Therapy and Cell Therapy Products
|
Published in |
Advances in experimental medicine and biology, January 2015
|
DOI | 10.1007/978-3-319-18618-4_3 |
Pubmed ID | |
Book ISBNs |
978-3-31-918617-7, 978-3-31-918618-4
|
Authors |
Anthony Ridgway, Francisca Agbanyo, Jian Wang, Michael Rosu-Myles, Ridgway, Anthony, Agbanyo, Francisca, Wang, Jian, Rosu-Myles, Michael |
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. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
India | 1 | 2% |
Unknown | 46 | 98% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Doctoral Student | 7 | 15% |
Student > Master | 7 | 15% |
Student > Ph. D. Student | 6 | 13% |
Student > Bachelor | 6 | 13% |
Researcher | 6 | 13% |
Other | 1 | 2% |
Unknown | 14 | 30% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 5 | 11% |
Biochemistry, Genetics and Molecular Biology | 3 | 6% |
Pharmacology, Toxicology and Pharmaceutical Science | 3 | 6% |
Chemistry | 3 | 6% |
Agricultural and Biological Sciences | 2 | 4% |
Other | 13 | 28% |
Unknown | 18 | 38% |