In 2020 only a few cell and gene therapies (CGT) are approved for commercial use, but hundreds of clinical trials are underway, and demand for manufacturing capability is set to grow. However the manufacturing equipment platform and associated digital capabilities are early in their maturity life cycle, in contrast to the established equipment platforms and digital capabilities for small molecule and biologics manufacturing. This is because there are many differences in CGT manufacturing that affect the IT systems requirements.
Personalized, made-to-order product requires a direct relationship with the patient and a very rapid turnaround time, with very dynamic scheduling. The end-to-end supply chain is orchestrated to make and deliver a batch of one for a single patient, which is very different to the way most lifescience products operate today. The batch is linked to the identity of the patient, its chain of custody needs to be closely managed and the logistics is on an individualized basis. Starter cells are much more variable than made-to-stock products. Outcomes need to be tracked over the long term in order to optimise processes and in some cases for outcome-based reimbursement,
For inventory-managed made-to-stock CGT products, the process and steps are often similar to existing biologics processes but there are some complicating factors. Raw materials are often expensive and difficult to source; the manufacturing process is more complex and immature; sometimes the storage conditions of the products are extreme (e.g. temperature) and shelf life is short, so inventory is difficult to build. Consequently the products can be expensive to buy which results in orders and deliveries being made on an individual patient basis, more like individualized therapies. Manufacturing might use similar or common technology platforms for these modalities but with different configuration and workflows through the systems.
The IT for Cell and Gene Therapy Workstream is a collaboration of a dozen leading biopharmaceutical manufacturers who are active in this area to develop and progress a vision and roadmap for digital capabilities to support cell and gene therapy manufacture. This has been expressed in a vision for digital capabilities for cell and gene therapy targeted to educate IT professionals of the distinctives of cell and gene therapies, so that they are productive faster and the solutions they create have better foundations, support more products, and are more maintainable.
Within that overall framework, the group has prioritized a set of topics to progress together during 2020-21. A key challenge is to identify a minimum viable architecture to support the range of manufacturing modalities within the CGT arena. Special thought is needed on the conflicting demands of CGT traceability and privacy, and how starter cell variability data can be captured, aggregated and analysed. Dynamic scheduling needs to accommodate patient needs while optimising utilisation of the equipment. And there are new challenges with integrating the types of equipment used for CGT manufacture, and some emerging closed systems approaches.