The time is now: why it pays to be prepared for the commercialization of advanced therapies

The impending commercialization of advanced therapies

Advanced therapy clinical trials are growing, with a record number of therapies being approved in 2021¹. This increase in approvals has led many to predict that the commercialization of advanced therapies is not far away. It offers the potential for fantastic breakthroughs for patients. But it’s vitally important that the supply chain is fully equipped to deal with this new reality. Pharmaceutical companies, suppliers, and manufacturers need to start preparing themselves for this demand right away, with storage facilities being a key concern. Failure to get ahead of the issue will increase cost and time pressures further down the line.

The need for cryogenic storage

The efficacy of a cell-based therapy is based on the number of viable cells, while the efficacy of a gene therapy is based on the number of intact gene constructs. For those therapies to remain viable, they need to be cryogenically frozen below -150°C – typically in liquid nitrogen (LN2) at -196ºC. At this temperature, cells can be stored for a near unlimited amount of time, but crucially, at least for the three to five years required for the typical shelf life of a pharmaceutical. Therefore, robust cryogenic infrastructure is required to ensure patients can receive treatment where and when needed. An optimal cryogenic infrastructure provides storage locations where therapies are needed, or at a place where they can be quickly and effectively transported to the point of care. Specialist storage technology monitors temperature to the individual package level, while innovative shipping packaging maintains and continuously measures the temperature and location.

The risks of not having such logistics in place are high. Clinical trial material is both scarce and valuable, and for advanced therapies, often irreplaceable. Temperature deviations can also have a significant effect on the efficacy of treatments. Properly controlled storage conditions are the prerequisite for a successful therapy and the patient’s safety.

Leading the way

There are people all over the globe who would benefit from advanced therapies. However, availability of these therapies is currently restricted to a few countries with the necessary infrastructure. World Courier is committed to creating logistics infrastructure in more places so more patients can access these life-changing treatments.

Currently, the countries with the most advanced scientific and medical research are leading the way in advanced therapies. The ability of individuals and health systems to finance these treatments is also crucial. The US, Europe, and Japan are important centers of excellence, while Singapore, Israel, and South Korea also make positive advances. Ethical and religious reservations and the related regulatory frameworks can play a significant role in the acceptance of advanced therapies, especially those based on stem cells or embryonic cells.

ICS_Brooks_3PL_Cryogenic_074_rgb_rtchd LR

The benefits or early investment

Advanced therapies require huge investment, and the same can be said of the logistics to support them, with costs reaching hundreds of thousands of dollars. Deploying tanks, fitting out the areas, and setting up equipment account for immediate expenditure, while ongoing running costs include equipment maintenance and LN2 consumption. Some companies may be tempted to delay investment until sufficient demand is generated. However, this can be a risky tactic. Staff training is a significant component of cryogenic logistics, and delaying this could hamper the progress of any prospective trials. Global supply chains have also been hit hard by the COVID-19 pandemic, which, as an example of an unexpected disruption, could make it more difficult to obtain equipment and materials in a timely fashion.

Preparing for commercialization now is likely to reap the rewards in the short term. Trained staff, fully equipped facilities, and robustly tested procedures being ready at project commencement will smooth the transition and strengthen relationships with clients. Quality and operational management systems should underpin the solution to ensure the highest standards. Those who are most prepared are more likely to be seen as reliable experts in the field. A lack of reactivity in this process hinders the ability to fully partner with drug manufacturers, which ultimately impacts patients' access to medicine.

A real-world example: Japan

In 2014 we started creating cryogenic storage solutions in Brazil when we deployed an area to store a DNA library that ran successfully for three years. By 2019, we made the strategic decision to invest in more cryogenic areas to cut deployment times in readiness to support our partner's development of advanced therapies. This initiative ran parallel with many other strategic decisions connected with our advanced-therapy transport offering as we predicted that future demand would rise.

In Japan, 2019, we explored various solutions to solve needs ranging from backup cryogenic solutions to active live trial supply chains. Our experts strategically deployed a modular solution aligned to projected demand, catering to small-medium volumes' storage needs, offering fully dedicated cryogenic tanks.

While deployment was being discussed, the first opportunity arrived, and we completed the final stages of implementation while our centralized team translated the clients' needs and strategic objectives into operational work instructions. This included a periodic report sent to the client, communicating updates on the project progress, and confirming they were ready to operate as soon as the tanks were qualified.

A rigorous change control process ensured that the cryogenic tanks performed as expected and that there was no risk to product quality, the patient, or the operator's safety. Some of the most relevant quality aspects included the qualification of the tank. This includes - User Requirements (URS), Installation Qualification (IQ), Operational Qualifications (OQ), and Performance Qualification (PQ), involving temperature mapping, as well as the development of related operational and technical SOPs.

Thanks to strong collaboration among our quality, operations and engineering teams, under the coordination of our project management office, we delivered the solution in just seven months. It was on time, on budget and immediately ready to receive the products, with a fully trained operational team providing support.

“Thanks to close discussions between our teams, we came to the ideal set-up for the facilities and equipment for storing cells. As a result, we are very satisfied with the solution provided by World Courier.”
World Courier client, Japan

The solution model was replicated in other markets and reviewed to incorporate new technology with a continuous improvement approach. The result is a replicable model for cryogenic storage solutions where turnaround time has been improved and can be deployed in 4-5 months, providing that supplies are readily available. Since then, we've expanded our advanced therapies storage capacity across the network, with Australia and Mexico going live in 2021.

Ready for the future of advanced therapies

World Courier is committed to providing future-ready solutions for its clients. We have doubled our cryogenic storage capacity during 2021 and will continue to expand our network in 2022. Cross collaboration between our teams and standardized procedures ensure networks are set up quickly and efficiently to meet the demand for emerging commercialization of advanced therapies.

To discover how World Courier can support your storage and transportation needs for advanced therapies, get in touch with our team of experts today.

[1] https://alliancerm.org/press-release/global-cell-gene-therapy-sector-on-pace-for-annual-records-in-product-approvals-and-financings-says-alliance-for-regenerative-medicine/