CGTP (Cell and Gene Therapy Products)
Cell and Gene Therapy Products (CGTPs) include somatic cell therapy products, tissue engineered products and gene therapy products as well as combined products.
Unlike biotechnology products which are mostly purified proteins of cells, CGTPs contain living and functional cells
The boundary-crossing nature of CGTPs are subject to a wide variety of regulatory oversights to encompass product development and administration in the clinical setting
The use of CGTPs posts some difficult-to-appraise risks, such as tumourigenicity, immunogenicity, in vivo migration of transfused cells and reversibility of administration in the event of an intolerable reaction
In addition to the requirements of current Good Manufacturing Practice (CGMP), the principles of current Good Tissue Practice (CGTP) need to be applied in the quality assurance of a CGTP.
The key benefits of regulating CGTPs separately under this framework will:
- safeguard public health and patient protection
- ensure the level of regulation applied matches the level of risk posed by specific products (tiered risk-based approach)
- provide a more flexible framework to respond to changes in technology
provide regulatory requirements that are unique to CGTPs which is cross-boundary in nature. Hence, call for integrated regulatory oversight for quality, efficacy and safety of the product
- reduce the ambiguity about what should be included or excluded from the regulation.
This framework lays down specific rules on registration and its data requirements [Chemistry, Manufacturing Control (CMC), nonclinical and clinical], supervision, risk management plan (RMP) and pharmacovigilance of CGTPs. Included are some regulatory procedures and guidelines in new areas such as current Good Tissue Practice (cGTP).
The cross-boundary nature CGTPs involves a multidisciplinary approach; therefore its full control will also be subject to various other regulations (authorities), hence an integrated oversight is imperative, as follows:
- The clinical use/medical procedure of the product will be under the ambit of Medical Development Division and Medical Practice Division of the Ministry of Health, Malaysia
- The device element of such products must comply with the Medical Device Act and regulations under the ambit of Medical Device Authority (MDA) of Malaysia, and
- PCB will ensure the medicinal product’s quality, efficacy and safety