March 18, 2022

Cell and gene therapies – new treatments, new value

In the new world of cell and gene therapies, what creates value? The obvious answer – the patient-specific therapy itself – is only part of the story.

At the recent Gene Therapy for Rare Disorders conference in Boston, I had the opportunity to discuss thoughts on value with an audience dedicated to delivering transformative gene therapy treatments to patients with rare, often debilitating conditions such as hemophilia, sickle cell anemia, or inherited vision disorders. Everyone agreed that gene therapies themselves are remarkable, and valuable in their own right. But if you can’t get that gene therapy to the patients who need it, or eventually get reimbursed for doing so, that inherent value goes only so far.

Comprehensive value in cell and gene therapies lies in both the treatment itself and the value chain that supports it – the full range of activities that make the product valuable. 

And that value chain often presents breaking points. Patients need to be qualified and approved for a gene therapy treatment, for example. Doses must be successfully manufactured, and then delivered to the intended patient at the right time – which is often much harder than it sounds. Successful reimbursement may require detailed long-term patient monitoring and data capture for a decade or more. 

Solving those value chain challenges goes far beyond having a product backed by proven science. Advanced therapies require advanced technology. High-quality data capture is a must-have at every step in the value chain, to provide transparency and traceability, satisfy regulatory requirements, and control COGS. Managing that data and the workflows within which the product moves requires a next-gen digital orchestration platform, so that you can make sure no opportunity for delivering one of these revolutionary products is missed.

At the conference, awareness of the importance of value chain data had clearly grown. In our value chain discussion, interesting questions were posed about all the potential uses of value chain data, and how this data could help with reimbursement. That’s a question that our sector is working together to solve, with new concepts such as value-based pricing. Many of these concepts will require sophisticated, ongoing data management.

It’s clear that the science behind cell and gene therapies has continued to advance at light speed ahead of the infrastructure and ecosystem intended to support them. No one single point in the value chain is yet fully prepared for gene therapy, and all the players in the ecosystem are still learning to collaborate effectively. At Vineti, we’re continuing to innovate on workflow orchestration and data management –we recognize its value and we look forward to a future of robust and insightful analytics.  Our PTM® enterprise platform is a critical solution for value chain challenges, and allows cell and gene therapy organizations to scale, even when they don’t know or can’t see the changes or obstacles in the value chains of today and tomorrow.

Louise Pacini is the Chief Operating Officer at Vineti. If you’d like to learn more about ourPTM® platform for cell and gene therapy supply chain orchestration, please contact us.