Although cell and gene therapies (CGT) are becoming increasingly popular and advanced, there are multiple challenges needed to be overcome before they become mainstream.
Participant responses to poll question posed in Pharmaceutical Technology/INTERPHEX webcast, “Editors’ Series: Overcoming Commercialization Challenges for Cell Therapies and Gene Therapies”, July 28, 2020.
Processing
Mass-producing materials for cell therapy require immense, meticulous, labor. It is an interactive process involving preparing and collecting samples, cell washing, and cryopreservation. Each step must be delicately executed as there is a high risk of contamination, especially if the labor is done manually.
GMP Regulation
GMP, or good manufacturing practice, is a set of practices that are required to be met by CGTs. This is easier said than done as CGTs also need to meet quick turnaround times. There is a connection needed to be formed between the manufacturing, or contract developer and manufacturing organization, and the logistics, or logistics provider, in order for the CGT to be considered GMP.
GMP also requires a reasonable manufacturing cost. There are currently two CAR-T cells, type of CGT, therapies in the market, one cells(get it?) for $475,000 per dose and the other $373,000. They cost over $100,000, 70% of which are spent on labor and GMP facilities. Unless these colossal expenses are reduced, CGT will not become mainstream.
Starting Material(Resources)
On top of the manufacturing costs, it is unsure if cell and gene therapy manufactures can mass-produce their supplies. If CGTs become mainstream, companies producing these drugs will immensely increase their demand from their manufactures.
Plasmid DNA is a material vital for both gene and cell therapies. Even without CGT being mainstream, the demand for plasmid DNA is increasing at a rate where suppliers are unable to keep up; according to VanCott.
Another problem is transporting the materials from different locations. From the harvesting location to storage, to a lab, etc. Experts consider using regional blood centers as a transportation network. They have a well established supply chain, effective method of transportation. With this being said, a standard process will emerge which is simple, safe, and robust.
Supply chain model for CGT
References