Autologous CGT is a catch-all term for a number of therapies that aim at providing breakthrough patient-specific medication. These therapies could be fantastic news to patients around the globe, but they pose a logistical challenge to the pharmaceutical companies, since they need to move away from traditional batch manufacturing and long lead times in order to enter the era of customized medication. Time for some insights on the specifics of the logistics process supporting autologous C&GT.

In recent years, there have been numerous new types of medication under the denominator Autologous Cell & Gene Therapy. These promising therapies are expected to improve or even save the lives of people with various diseases. The key idea behind Autologous C&GT is that you develop a customized, patient-specific therapy by altering a number of the patient’s cells. This treatment has the potential to deal with certain metabolic diseases, but also certain types of cancer. For Autologous Cell & Gene Therapy, the patient is the starting point of the whole process and also the starting point of the Supply Chain. In other words, the patient is the source of the genetic material that will be used to develop a treatment.

These treatments are very interesting and promising, but also quite challenging from a logistical point of view, as these new advanced therapies bring new challenges on different levels that pharmaceutical companies need to tackle. It really requires new integrated Supply Chain processes across the plan, source, make & deliver phases within a supply organization. Each function has a significant role to play, and each step in the process requires a specific approach: from complex cold chain distribution to patient-specific planning & scheduling. Clear, end-to-end communication and an agile Supply Chain set-up are of utmost importance in this specific domain of Life Science.

Keeping track of the customized medication

The main difference between Autologous Cell & Gene Therapy Supply Chain and Traditional Therapy lies in the source of the medication. For Autologous C&GT, you could say that the Supply Chain is a loop that starts and ends with the patient. Between the moment of apheresis - collecting cells from the patient - and providing the treatment to the patient, multiple hand-overs take place. It is clearly important to keep track of the product itself throughout the end-to-end process, because it is patient-specific. In each of those hand-overs, the so-called Chain of Identity and Chain of Custody must be maintained at all times, in order not to mix-up samples and prevent mistakes.

Throughout the entire Supply Chain, the Chain of Identity and Chain of Custody are maintained at every step.

Monitoring and tracking the whole Supply Chain is one of the most critical aspects in Autologous C&GT. It is necessary to exactly know where each sample is located, so that the patient receives the right therapy. From a Supply Chain perspective, this requires pharma companies to completely rethink how they do things. The Supply Chain is handled individually, serialized and not in a batches, as is the case for many other product lines.

The biggest challenges

One of the biggest challenges within Autologous Cell & Gene Therapy is that timelines are much shorter than for other (generic) compounds. For example, the timelines between sample collection and cryopreservation are very limited – often only 24 or 32 hours. This puts enormous pressure on the distribution network, as even the slightest deviation can render patient cells unusable, which means that irreplaceable source material could be destroyed.

Another logistical challenge are the frozen samples. These samples are already cryopreserved and sent in specific shipping containers, filled with liquid nitrogen, which poses certain safety risks and requires specific transportation methods. But there is more:

• Batch versus serial

In the traditional pharma Supply Chain, units are produced by the thousands in one batch, while in Autologous C&GT the Supply Chain is individualized, tailored medication for one specific patient. Each sample is followed up individually. Increased volumes therefore require customized Supply Chain designs and innovative strategies.

• Traceability and systems

Legislation by region and/or per country is very important within the pharma world, and especially so with regards to Autologous C&GT.

Europe has a different regulation than the USA, Africa or Asia, but The CoI or CoC must be respected across the board. Each step and sample must be documented in detail. These extra requirements clearly have an impact on the IT-systems as well. ERP systems or Supply Chain Planning Systems are generally not designed to support the specific data requirements and the typical milestones that can be seen in this specific Supply Chain Process. It requires end-to-end system thinking to be able to adequately redesign the IT-systems, in order to support the whole process. Not a small task.

• Lead times

The Supply Chain context of Autologous C&GT is completely different from that of traditional pharma Supply Chain, which is characterized by batch manufacturing and sometimes very long lead times. The logistics for Autologous C&GT, however, require very short lead-times, given the nature of the illnesses and the therapy.

Lead times between the collection of patient genetic materials, medication development and administration of the medication are kept extremely short, compared to traditional pharma Supply Chain. Each step in the process needs to be monitored closely. The so-called ‘Vein-to-vein time’ captures the timeframe between cell collection and dosing. Keeping in mind the patients’ disease progression in between, it is critical to reduce the ‘Vein-to-vein time’ as much as possible – putting pressure on the distribution network to transport samples at record-breaking speed.

• Cold Chain and temperature control

Autologous C&GT requires very strict temperature monitoring of a combination of two condition types: Refrigerated Shipments (2-8C) and Cryoshipments (-190° C) from the hospital or apheresis center to the processing center. The challenge for cryoshipment is that samples are shipped in a mobile cryoshipper at -190°C and that it has an impact on the maximum duration of the shipment. Other challenges include scalability of the shipper fleet and possible outsourcing, distribution, handling of liquid nitrogen and maintaining the CoI and CoC in every hand-off.

• Milestone Planning

Autologous C&GT requires very strict Milestone Planning, in order to make sure that the tight lead time schedule is realized time and again. Milestones typically mark critical decision points, the completion of major steps and milestones in the patient journey. To keep track of all of this, pharmaceutical companies need to look into adjusting their IT-systems, and their organization in general. It takes specific roles and attention to make it all happen without mistakes, within set time limitations. Everything needs to be more interconnected.