Updated December 2019
As cell and gene therapies continue to expand, researchers increasingly require reliable logistical support. The safe and efficient transportation of cell and gene therapy products is now critical to patient care and manufacturing efficiency.
In the last five years, more than 1,000 clinical trials have used cell and gene therapies. Furthermore, over 500 cell and gene therapy candidates are being actively researched (1). Indeed, the 2017 approval of two autologous CAR T therapies has clearly shown that for treatments like this to expand, the paramount logistical support has to evolve rapidly.
The cell and gene therapy sector, which could deliver life-changing treatments for some of the world’s most severe conditions and diseases, relies upon a logistical supply chain that must perform to high clinical standards.
Often, cell and gene therapies also require high harvesting of patient tissue that must then be transported to the manufacturing facility, with the resulting product returned to the patient’s clinicians. As the patient is an integral component of the supply chain, the logistical support provided must be robust and secure. Any breakdown or erosion in the efficiency of the supply chain could have massive consequences for patients.
Cell and gene therapy also requires high levels of logistical support to deliver each therapy to individual patients. In both scenarios, traditional cold supply chains had to evolve to meet the technical needs of laboratories and manufacturers alike.
As cell and gene therapy is a new branch of medical science, logistical services need to offer transparent chains of custody bespoke to the individual therapy and patient. Currently, manufacturers and therapy owners are working with specialist logistical service providers to develop the supply chains they need to deliver their therapies to clinicians.
Building a supply chain to support a cell or gene therapy needs a clear understanding of many components. The therapy must be available when the patient requires their treatment, but doing this may also require swift, on-time delivery of starting materials to provide the manufacturing facility with the optimal product while meeting manufacturing run commitments.
Here, timely and reliable deliveries are critical components of the supply chain. A definite challenge for logistical support services is contingency planning. Communications when an incident occurs are critical at this stage to ensure contingency responses are implemented efficiently.
Gene therapies in particular require bespoke logistical support that can include new packaging innovations, new storage options, and validation of the cold supply chain. This ensures the integrity of the therapy is maintained as it travels through the supply chain from the manufacturer to the clinician and, ultimately, their patients, facilitating use on arrival in the clinical environment.
The supply chain for cell therapy can become highly complex. It is not uncommon for several different cells that need collecting from multiple sites to be delivered to the manufacturer, with the resulting therapy then returned to the patient. The challenge is to understand each component of this supply chain and its individual needs.
A patient’s apheresis collection may need refrigeration at between 2-8°C. Moreover, finished therapies may need liquid nitrogen shipment or dewars at cryogenic temperatures. In this scenario, the logistical support needed is manifold and must integrate into a supply chain that can support the precise needs of cell and gene therapy.
Challenging cell and gene supply chains
Currently, logistical support for the burgeoning cell and gene therapy sector requires the designing of bespoke support services. As this industry is relatively new, standardisation has yet to develop across the supply chain. However, logistical support service providers will be able to develop some standardisation as therapies expand and become more commonplace.
A good example will be when a drug trial moves out of its Phase 1and 2A stage to 2B, as this usually means the sites where the trial takes place expand. When a trial becomes multisite, it is crucial that the logistical support the trial has can easily scale without a reduction in the quality of the logistical services supplied. There will always be a requirement for bespoke logistical support carefully crafted to the need of the product. However, this will be joined by some standard practices driving efficiencies. The key is in the understanding by the logistics provider where these efficiencies can be made without risking quality.
The packaging that cell and gene transportation requires is also an important consideration when developing reliable logistical support for trials and gene lab support. When international shipping is needed, it may not always be possible to obtain fast turnarounds (24 hours) between countries. The ability of the packaging to hold its temperature variance within stated tolerances can be challenging, especially when opposing climatic regions may need to be considered in a single shipment. Furthermore, the physical touchpoints that exist at a cell or gene’s final destination can often be highly variable. Storage before patient administration needs to be carefully considered to ensure consignments can withstand any potential delays when they arrive on site. The challenge for logistical support service providers is to become more involved in the design of the end-to-end processes to ensure they understand and can support any variances in these clinical touchpoints.
When designing a logistical supply chain, seeing the chain as a whole and not as individual components is vital. Making a change to one component of the chain can often be counterproductive, as the knock-on effect of this modification has not been thoroughly thought out and tested. Here, the interdependencies of supply chain components must be appreciated.
As a therapy moves into the manufacturing stage, the logistical support needed can be critical to success. The developers of the therapy should consult with logistical support service suppliers regarding the location of manufacture sites, which will be centralised in the first instance, but will later need co-location support as the therapy rolls out to patients on a global scale.
Logistical support also needs careful consideration when fresh versus frozen starting materials for therapies are deliberated. The supplier of logistical support can advise how fresh or frozen manufacturing could impact on scheduling, delivery, and which is appropriate to maintain the potency of the therapy concerned through the chain of custody.
Personalised logistics and specialist couriers
As cell and gene therapies expand, the need for logistical services also rises. The challenge facing logistical specialists is supporting the myriad of trials and therapies that enter the market as both autologous and allogenic therapies increase. A therapy-specific risk assessment is clearly vital to carry out when identifying the risk areas within a logistical supply chain.
Personalised medicine is a massive growth area. The quantities of tissue that need to be transported will grow exponentially as personalised therapies become more commonplace. Some form of standardisation will be possible, for instance, with packaging, but the logistical support and setup needed remains bespoke for each therapy.
Tracking technologies also become increasingly important as logistical services expand. The already-existing tracking technologies that utilise GPS will become more sophisticated. Here, the arrival of the 5G cellular network enables logistical service suppliers to expand their services, as the fast and more efficient digital network can be leveraged to enhance the logistical processes available to users. These new mobile services will provide efficient and accurate tracking of consignments as they move between regions that may have very different compliance regulations to support.
Undoubtedly, cell and gene therapies have the potential to transform the treatment of many diseases and conditions. As this branch of healthcare expands, it requires a support network to ensure it can deliver professional and effective patient care. One key aspect of this care is the logistical support that these therapies rely upon.
Understanding the particular needs of these therapies and developing bespoke supply chains to meet these requirements will see existing logistical suppliers expand their services aimed at specific sectors. With a background in cold supply chain logistics, they will build on this knowledge to support the burgeoning cell and gene therapy sector.
The key challenges will be developing processes that can be applied to what can be highly focused therapies and patient groups. Evolving the tracking of tissues, cells, and resulting therapies as they travel through the supply chain (which can include traversing multiple regions and manufacturing processes) is also a key challenge for the logistics industry.
The growing cell and gene therapy sector will need comprehensive and integrated logistical support that is tailor-made for each therapy. The interdependences of each supply chain component must be clearly understood with appropriate contingency plans in place. More input at the early stages of cell and gene therapy development is also vital to ensure logistical support service providers have sight of what is required to deliver potentially life- saving treatments to patients.
Ultimately, logistical support service providers have to become more intimately involved with the clinicians, labs, and manufacturers they are serving. This will help them design the logistical support they need to deliver the therapies or patient care they are developing.