Breaking the Bottleneck of Affordability

Advances in cell and gene therapies offer the potential to transform medicine. But current pricing structures are unsustainable – and they are severely limiting the introduction of new, potentially life-changing or life-saving therapeutics. 

Though research and development in this sector is moving at an impressive rate, the complexities of scale for growing, sourcing and transporting cells and other components of cell and gene therapies are evident – and this is clearly feeding into costs.

Part of the solution to the cost conundrum when treating patients is point of care automation – particularly, closed automated systems for processing cell therapies. The introduction of closed systems allows the services to be delivered from a less expensive (lower grade) clean space, requiring fewer and lower-skilled workers. Equally, these developments mean that multiple systems can co-exist in a single area. Once you have real-time quality-control testing at each different stage in the process, for instance, in taking blood delivery, isolating, propagating, testing, and harvesting, the time to deliver a product back to a patient can be reduced.

However, processing and technology are only one part of the problem. The business structure for developing and commercializing cell and gene therapies also increases costs (potentially unnecessarily in my view). The typical lifecycle of a new therapy begins with a research institute or group producing a potential new treatment. Often, in this sector, this research is being undertaken at a research-intensive hospital. The new therapy is then developed in house for as long as is feasible, before ultimately being either spun-out into a traditional biotech company. Further development – fuelled by intensive fundraising – is then followed by recruiting the services of a commercial CMO. This process is costly at all levels and erodes the core value of the treatment as ownership is diluted as development progresses.

But what if research hospitals could keep things in house, and fully develop and process the products themselves – even taking products all the way to the patient? 

Typically, hospitals either don’t have the capabilities or the expertise to work to the GMP standards required for cell and gene therapies, which are highly regulated very specialized. In some cases, where hospitals have facilities, they are simply not able to make use of them as they do not have staff with the required know-how. Notably, this latter point is certainly not a criticism of the hospitals but a reflection on the technically challenging nature of the process.

An exciting alternative would be to build a network of hospitals around the world that are equipped to develop and process cell and gene therapies. And that is something Orgenesis is creating.

The proposed consortium would evolve a network or a partnership model, where external experts would be brought in to set up and run a series of hospital-based GMP facilities on behalf of the host hospitals. The model gives incoming experts direct access to onsite researchers, where they will see the new and innovative research programmes that are ongoing and be in a position to assist in turning these treatments for direct patient care at the hospital site. 

If the expertise was derived from a single external source – ideally an established specialist in the field with the capacity to take on such a wide-reaching project – it could become the overarching “management company”. This entity would bring together all the capabilities of the network hospitals, streamlining the whole program and providing a commercial window into the services for external clients, as well as for the research and development groups within the consortium.

The concept is akin to the Uber model. Uber gives drivers the technology (a standardized app) that allows them to work as a taxi driver, using their own car. Here, the specialist entity would provide the technology (licenses for closed automation systems and streamlined, specialist processing services and treatment therapies) to hospitals so that they can develop and commercialize advanced therapies. The key objective is to ensure that the same high standards of processing are maintained throughout the network – using harmonized, automated systems for patient treatment and care throughout would be key to success in this regard. 

Hospitals would clearly benefit from this model by retaining ownership and the value of the product. And as that value is retained, rather than being diluted through the development and processing methods, overall costs will be lower, and therapies should be cheaper. 

I also believe that large biotech and pharma companies would benefit from this model. If a hospital were to approach such a company and ask whether they would like to take on the marketing license for a new therapy that the hospital is processing, to a high standard, as part of an established network, I’m sure they would be delighted.  

Overall, I believe this model, if integrated with modern, automated, closed systems at the point of care for treating patients, has the potential to reduce the currently overbearing costs associated with taking cell and gene therapies through to market.