Cell and gene therapy manufacturing may never be standardized but the whole industry would gain if firms collaborated to develop common methods for some processes according to an expert.
Manufacturing cell and gene therapies is an expensive business, partly because no two products are made the same way.
A recent study in the journal Nature suggested the average cost of making an autologous cell therapy is between $100,000 and $300,000 per patient.1
The authors attributed the high cost to the use of novel and specialized manufacturing processes [which] make scaling to meet commercial demand a significant challenge for all.
A separate study in the Journal of Clinical Oncology also concluded that difficulties scaling-up the bespoke manufacturing processes and technologies used to make cell and gene therapies significantly increases production costs.2
Market tensions
And high costs are a problem, according to Maria Whitman, managing principal at consulting firm, ZS Associates, who said cell and gene therapy firms need to find more economic ways of making products.
Standardization in manufacturing across the industry is not likely to be the priority for standardization in the short termHowever, the in-market cell and gene therapies have illuminated a number of tensions in the U.S. healthcare system which was designed for pills and biologics.
With over 200 CAR-TCR trials alone in the United States, there is need for standardization of aspects of the process to enable scale and commercial viability of these technologies. The challenge is that, today, each manufacturer is in part by necessity establishing their own process and protocols, she said.
The key is to look for similarities in processes, according to Whitman.
Potential areas for manufacturing and logistical standardization include apheresis protocols, labeling and information management, tracking processes, and training certifications, she said.
Whitman suggested contract manufacturers could help to identify common manufacturing challenges if customers are willing to work together and share information about noncompetitive areas of production.
The process question we should be asking as an industry is this: what is really competitive IP, and what is not? If we answer that, we can identify and solve for more systemic needs.
Logistics is another area where standardization would benefit the sector, Whitman added, citing developers of autologous therapies as the obvious example.
Autologous cell therapies are produced from the patients own cells. Typically the cells are harvested at a clinic and transported to the manufacturing facility before being returned to the patient. Ensuring such therapies are delivered in a timely fashion is vital.
According to Whitman, Manufacturers are trying multiple approaches to streamline the logistics of distance between manufacturing and patient administration. Some are developing in-house solutions and technology or leveraging partnerships to minimize risks and timing.
There is also a new industry emerging of companies forming to solve specific issues including apheresis networks, product manufacturers, as well as companies that create ordering portals, supply chain management systems.
One approach is to localize manufacture. Whitman said, There are already a number of manufacturers working on technologies to make point-of-care cell therapy a reality. Some academics are also creating their own CAR-TCRs, for example, and running trials in parallel with traditional manufacturer trials.
Ultimately the growth of the cell and gene therapy sector will depend on manufacturers ability to balance production and logistics costs with product prices. And the desire to find such a balance is clear, Whitman said.
Manufacturers will look for ways to optimize and automate the process where possible to reduce the cost of skilled human labor and continue to remove risk and drive efficiency in the system.
References1. http://www.nature.com/articles/s41434-019-0074-72. hascopubs.org/doi/10.1200/JCO.18.02079
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IP or Not IP: That Is the Question for Cell and Gene Therapy Sector - Genetic Engineering & Biotechnology News
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