By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1
In Part 1 of my conversation with Ryan Bartock, Head of Supply Chain and Network Strategy at Spark Therapeutics, we covered best practices for pharma, bio executives, and CMOs to successfully manage and even streamline the supply chain. Here, in Part 2, Bartock addresses the unique demands of a gene therapy supply chain.
What are the unique demands of a gene therapy supply chain?
Bartock: With the success of LUXTURNATM (voretigene neparvovec) and other high-profile product candidates coming to both the clinic and commercial market, there is no question that the field of gene therapy is growing rapidly. These novel products bring the potential for one-time, durable treatments to patients but also come with their own unique set of challenges:
- Accelerated regulatory pathways are becoming the norm rather than the exception. This generally requires commercially viable supply chains to be in place at the inception of a combined phase I/II program.If changes to the manufacturing processes, analytical methods, and/or supply sites are required later in the lifecycle, this can add complexity when scaling supply chains to support growth in the product or portfolio.
- Most gene therapy companies start small, often emerging from academic and other collaborations.As a result, supply chains often have a high degree of reliance on CMOs along with single sourced novel materials, reagents and analytical vendors. This introduces both complexity along with risk to supply performance and security.
- Viral vector development and manufacturing is critical to the gene therapy supply chain and competition for capacity and starting materials to produce them is increasing.While conventional controls like inventory and capacity buffers can be employed to buy down risk, this comes with an associated cost that will only increase as lead-times extend and capacity becomes more constrained.
- Because we’re bringing a novel medicine to the patient, often in a setting that requires either infusion or a surgical procedure, demand will remain volatile and capacity planning can be impacted by:
- Patient enrollment, screening failures, treatment cancellations/rescheduling
- Broad differences in demand across therapy areas (i.e. rare disease vs. more prevalent targets)
- Uncertainty in uptake rates as supply extends across global geographies.