Avoiding Catastrophe: Addressing the Supply Chain Risks in Cell and Gene Therapy Development

Thu, Nov 21, 2019 | By Phil Wilson, Sr. Vice President, Global Logistics and Supply Chain

There is no question that the groundbreaking treatments being developed in Cell & Gene Therapy (CGT) are fundamentally changing the way patient care is managed around the world. As with most revolutionary breakthroughs in medicine or any other industry, the supply chain is critically important. These revolutionary therapies only have value if you can effectively get them to the patients, or in the case of CGT, into the patient’s vein. Furthermore, CGT treatments are extremely fragile and require weeks to develop in multiple facilities. It cannot be stressed enough that a fail-proof supply chain is necessary.

The pharmaceutical supply chain is already one of the most complex in the world and rightly so; to ensure that the pills, vaccines or liquids have not been adulterated and are safe for consumption, every aspect of the supply chain must be vetted to ensure quality and integrity. CGT takes this to a whole new level. For conventional drug development, a multitude of qualified suppliers work with manufacturers to bring a product to market in a robust and vetted GxP process that has resiliency and redundancy. Risk is mitigated through multiple suppliers and manufacturers around the globe in redundant supply chains. If a batch of gel caps doesn’t pass final QA release because they sat on a hot tarmac too long, they can simply be discarded, and another will quickly take its place. There is no such luxury in CGT development. In the CGT process, one treatment for one patient can easily exceed $400,000. The patient is the supplier in many cases, and the margin for error is nonexistent. A patient’s life may literally hang in the balance.

From a logistics standpoint, CGTs are extremely sensitive to variations in temperature, requiring very strict controls. From the time of blood withdrawal to patient dosing, every aspect of the supply chain must be actively monitored and controlled. Time, temperature, and product security are just a few of the critical elements that need to be constantly monitored. Each stage of the supply chain plays a critical role in determining treatment effectivenss. To ensure success, a complete and vetted shipping system must be used.

CGTs are most often transported in a cryogenic state of liquid nitrogen contained in a vacuum dewar. On the surface, this sounds simple – it's a process and technology that has been used for decades. However, when transporting a single treatment with a value in the hundreds of thousands of dollars, the process needs to be much more robust. A shipping system should include the following elements: validation and shock absorption through advanced internal and external packaging that exceeds ISTA standards; state-of-the-art dewar technology and revalidation processes that ensure cryogenic holding times are actively monitored and confirmed for every shipment; active shipment level tracking that is based on redundant GPS, cellular and WiFi networks constantly monitored around the globe; and 24/7/365 support personnel who can intervene in emergencies. No exceptions. These are the standards that must be adopted globally to ensure patient safety and CGT success.

Currently Cryoport is using our years of industry experience and advanced technological expertise in helping set these standards with the governing bodies across the globe. Watch the video below to learn more about Cryoport’s proactive role in creating the critical guidelines necessary to develop and distribute regenerative medicine. 

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