I had the opportunity to write an article on the unique challenges of cell and gene therapies supply chains for our sister print publication, Life Science Leader’s 2019 CMO Leadership Awards Special Edition, which will hit mailboxes in March. Be sure to look for the article and the issue when it hits the streets. In the meantime, here’s a sneak peek at just some of the original data you’ll read in the forthcoming article.
For the official article, I conferred with three of the field’s most valuable luminaries representing both academia and industry — Dr. Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy at University of Pennsylvania; Simon Ellison, ISCT Commercialization Committee; and Ryan Bartock, Head of Supply Chain and Network Strategy at Spark Therapeutics — to determine how CMOs can and should successfully manage cell and gene therapy supply chains. Here’s a portion of Levine and Ellison’s insightful responses.
What are the unique demands of cell and gene therapies’ supply chain?
Levine: A large proportion of our reagents, materials, equipment are single source. Therefore, when there is a disruption in the supply, it can have a catastrophic effect on the ability to manufacture the therapy. With the growth of the field over the past few years, there are beginning to be more options for alternatives. Still, these must be validated, and demonstrated to be comparable to the primary materials in use.
Ellison: Traditional pharmaceutical supply chains differ between clinical and commercial scale. Advanced therapies have essentially the same time critical & temperature sensitive supply chain throughout their lifetime. In addition, this is the first time that the patient has been part of the supply chain, either donating and/or receiving a live therapy.
This puts pressure on the supply chain and means that companies must take a Logistics by Design approach and create logistics platforms that can connect therapies to patients at both clinical and commercial scale.
What advice do you have for pharma, bio executives, and CMOs to successfully manage the unique demands of the cell therapy supply chain?
Levine: First, know your suppliers and establish the relationships that will serve well in event of a disruption. Secondly, engage in horizon scanning for new technologies and suppliers for alternative materials. Thirdly, as an academic institution, we have established relationships with peer institutions that may be able to lend a material in short supply in an emergency supply situation.
Ellison: Advanced therapies are moving from first-in-human studies to commercial launch in around 5 years. This means that senior executives need to develop a vision of what their logistics platform will look like at commercial scale, whilst still in early clinical trials. This enables them to take a risk-based approach to developing their logistics platform. This approach enables the identification, and ranking, of Critical Logistics Attributes that can be addressed as they progress through the clinical development process. Giving them a functioning logistics platform as they come out of their pivotal trials
Decisions made early in the development process can impact on a therapies ability to commercialize. For example, if the donation or therapy is shipped “fresh” this is expected to give a better cell viability, however it creates a shelf life issue. Meaning that the shipping window is constrained by the duration that the cells remain active, etc. A constrained shelf life means that the therapy can only be collected/distributed within a certain distance from the manufacturing center, and therefore the developer will need multiple manufacturing sites. This brings cost, regulatory, and manufacturing equivalence issue.
Having a fresh product is a perfectly viable commercial strategy but the therapy developer needs to understand the impact early in the development cycle and either budget for process development to create a cryopreserved supply chain, or the impact of multiple manufacturing sites.
In other words, streamlining the supply chain is reliant on a Logistics by Design approach and planning with the future in mind.