In his recent article, “The Last Mile in Beta-Cell Replacement Therapy for Type 1 Diabetes: Time to Grow Up”, Lorenzo Piemonti delivers a interesting call to action: we must rethink how we develop advanced therapies—not just for technical success, but for real-world scalability and affordability.
A key takeaway is his advocacy for Quality by Design (QbD)—not just for product quality, but for delivery feasibility. What does this mean in practice?
Instead of designing advanced therapies for ideal conditions—complex, expensive, and hard to scale—we should aim for solutions that are “good enough” to work safely, effectively, and affordably for many.
Designing from the outset with the end-user and system in mind: simple administration, minimal infrastructure requirements, and realistic cost targets. Imagine beta-cell therapy that’s thawed at the bedside and delivered with a standard syringe—no operating room, no GMP suite.
It also raises key questions for the scientific community:
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How can we integrate economic and operational feasibility earlier in therapy design pipelines?
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How do we avoid creating therapies that are “beautifully designed but inaccessible”?
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How do we define “good enough” in ATMP development?
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Can we incorporate these trade-offs earlier in design?
Let’s discuss!