March 25, 2022

Gene therapies – simpler doesn’t mean simple

Cell and gene therapies are produced and delivered via the most complex workflows in the history of medicine. Among those workflows, though, there’s a range of complexity. Some of these transformative treatments, such as CAR-T cell therapies for cancer, are produced from a patient’s own living cells. Others, such as gene therapies for rare inherited disorders, can be batch-produced up front and then paired with the right patients.

That difference between “Batch of one” and bulk production can create an impression of simplicity when it comes to gene therapies. If one doesn’t need to collect and manage living cells to make a gene therapy, then these therapeutics must be easy to manage.

But as I’ve learned from conversations with biopharma experts and industry gatherings such as the recent Gene Therapy for Rare Disorders conference, simpler doesn’t mean simple. Gene therapies have their own inherent workflow complexities, and still require advanced approaches to workflow management.

That’s because 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. 

In gene therapy, that value chain often faces breaking points. Here are just a few that gene therapy experts have shared:

  • High-cost nature of the product: gene therapies are often significantly more expensive than most other pharmaceutical products. When a treatment costs $1 million or more per patient, each vial of product becomes extremely valuable. Keeping tight control of inventory becomes particularly important.
  • Multi-dose scenarios: to achieve efficacy, it is increasingly clear that many emerging gene therapy products will require redosing of patients, maybe throughout their lifetime. Each of these high-value doses will need to reach the right patient, and be closely controlled and tracked along the way.
  • Higher risk of patient-product mismatch or fraud: many gene therapies are dispensed in doses that are personalized to the patient using factors such as patient weight. Sending the wrong-sized dose to the wrong patient by accident could trigger an adverse event. Pharma experts say there are also greater risks of fraud related to entry of deliberately incorrect patient data
  • Long-term follow-up: as with other cell and gene therapies, regulators require long-term patient monitoring to understand safety and efficacy over time. New reimbursement models, including value-based or milestone-based systems, will also likely require long-term patient tracking and reporting.
  • Complex COI/COC: Maintaining full visibility of the patient journey may be even more complex than for cellular therapies. Gene therapy intermediate and finished products will likely be stored at a mix of internal and third party distribution centers, specialty pharmacies, or hospitals. Maintaining visibility of these expensive treatments will be challenging.

So while gene therapy may look simpler than a cell-based product, it’s still not simple. It’s increasingly clear that gene therapies will need their own version of automated Chain of Identity (COI)  and Chain of Custody (COC) – the two cornerstones of traceability and orchestration in cell and gene therapies.

Effective COI/COC requires capturing a high volume of data along the end-to-end product journey, often from dozens of touchpoints and myriad events that have to be seamlessly orchestrated.

In products delivered via multiple doses per patient. COI/COC and data must be maintained for each dose, tied back to the relevant patient, and tracked over the course of long-term follow-up. Even when a gene therapy workflow looks simpler than a CAR-T cell therapies, manufacturers are realizing that they’ll still need to manage complex workflows and data at both the start and completion of each patient and product journey.

Marc Puich is Senior Vice President of Business Development at Vineti, and previously spent a decade working in the field of pharmaceutical manufacturing systems. If you’d like to learn more about our PTM® platform for cell and gene therapy supply chain orchestration, please contact us.