The concept of cell and gene therapy (CGT) is not just a marketing slogan. With stem-cell research, CGT continues to make great strides as a weapon against cancer and other diseases. CGT is actually expanding at an unsustainable rate, with more than 1,000 clinical trials scheduled to take place globally by the end of 2019. Financing demand has also increased with stem cell research. Up to 20 cell and gene therapy products are expected to be approved by the FDA by 2025, after $7.4 billion was invested in the field in 2018.
The CGT initiative is making progress, but many challenges remain for healthcare professionals, researchers, governments, and businesses to overcome. CGT activity has proliferated in unprecedented ways, resulting in these challenges. It is unsustainable to continue as it is, and the system is stifled. Addressing these challenges can pave the way to clearer directions in pursuing new discoveries.
Let’s explore these challenges in detail.
The CGT system is currently designed for handling small molecules and biologics. To develop CGT at a large scale, it is vital to rethink the entire process from start to finish. Today, we face a number of challenges, including:
To identify and treat the appropriate patients that match with the research.
Around two out of every three clinical trials fail to enroll their intended number of participants, whereas about 20% of trials examining cancer patients do not enroll enough participants. Moreover, unlike traditional therapies, CGT calls for significant patient involvement throughout the long phase of treatment. CGT clinical trials will need to enroll more than 100,000 patients in the next few years, out of which approximately 15% of those patients may not be able to complete the trial at all.
Developing an effective treatment center model.
There is an inadequate level of management of cell therapies in many clinical practices. Clinical research facilities where clinical trials are conducted need to be accredited by organizations such as the Foundation for the Accreditation of Cellular Therapy (FACT) in order to meet the standards, however, the institutions in the US accredited by FACT are few in number.
The process of gathering raw materials.
According to the FDA, cell extraction and handling must be performed following Good Manufacturing Practices, which is challenging to apply to a large scale. Cell labs face challenges related to storage space and staffing shortages. Some labs are also incapable of following the instructions of their sponsors. Moreover, the health information technology infrastructure of many specialty centers is usually incompatible with that of other treatment centers.
Choosing and coordinating specialized couriers for cell delivery.
Most courier services lack the capabilities and expertise required to deal with transportation under refrigeration, freezing, and cryogenic conditions. Package and logistics providers have very limited capacity today as they handle a minimum of two shipments per patient and work with hundreds of manufacturers globally.
The challenge of ensuring patient safety, as well as reducing manufacturing costs, also must be addressed. Clinical trial subjects and commercial patients are at increased immunological risk because of the transformation in cellular mechanisms. On the mechanical front, most CGT efforts are being led by startups who lack the resources to gather essential data. To manage the production of the cells, the company relies on contractors and manufacturers. The problem is that potential partners are scarce, with waiting lists running months and even years.
Taking the next step
In the CGT space, we are seeing two key developments that will help overcome the challenges facing the industry. We must first decentralize our operating model. This will lead to the emergence of several types of businesses. To participate in this new economic model, businesses can build their capacities and enable decentralization or join forces with other businesses.
As the new healthcare ecosystem matures, its traditional linear and centralized operating model will be replaced by a decentralized model with hubs and spokes, allowing greater flexibility in where product development happens, where cells and tissues are collected, and where treatments are given to patients. Decentralized hubs and spokes will allow value chain partners to increase their capabilities and to adapt their roles. As a result of the network of local hospitals, access to medical care will improve, as will the ability to streamline patient referrals reduces the leakage of patients.
Change and opportunity
Even though we have made great strides in diagnosis and treatment, the clinical paradigm and drug development are not much different from that of the past. Personalized medicine, which now receives significant investments in healthcare, was not designed to deal with the vast growth of potentially curative medicine.
As CGTs are developed and commercialized, they pose a significant challenge to the healthcare ecosystem and have created significant structural obstacles to the delivery of potentially transformative therapies. It is an industry undergoing unprecedented change, which presents chances for existing companies to rethink their business models.
Decentralized business models and customer-centered value chains are similar to those seen in assorted consumer industries. Hub-and-spoke systems and patient-centered care will change healthcare forever. A network of treatment centers with advanced technologies will facilitate the collection of large datasets that will accelerate the development of more customized CGT.