- A coalition of drugmakers has agreed to work with Vineti, a San Francisco-based technology company, to usher in new standards that could ease some of the safety and logistic concerns surrounding advanced medicines like cell and gene therapies.
- These medicines often pass through complex networks of manufacturers, transporters and healthcare providers before they're delivered to patients. As many of them are personalized and meant to treat very ill patients, it's critical to know exactly whom each therapy is for and where it is along the supply chain. And yet, modern cell and gene therapies still use the same patient identifiers as more "legacy" treatments like bone marrow transplants — standards that, according to Vineti, "do not always cover all the use cases presented by today's personalized therapeutics."
- Vineti wants to establish a new, flexible patient identifier akin to a digital ID badge that could work for all types of advanced medicines and improve traceability, control costs and better ensure patient safety. The company now has more support, too, from that coalition of drugmakers, which includes several biotechs as well as large pharmaceutical firms like Novartis and Takeda.
The cell and gene therapy field hit major milestones over the last few years. In the summer of 2017, the Food and Drug Administration approved a first-of-its-kind cellular medicine from Novartis for a hard-to-treat form of leukemia. Less than two months later, another cell therapy developed by Kite Pharma was cleared for patients with a different blood cancer.
Before the end of that year, Spark Therapeutics received the first-ever FDA nod for a gene therapy targeting an inherited disorder. And since then, a small crop of advanced medicines developed by Kite, Juno Therapeutics and AveXis have come to market.
These successes have, in turn, enticed more companies to invest in cell and gene therapy research. Gilead, best known for its work fighting viruses, dropped $12 billion to buy Kite right before its first treatment got approved. Novartis then agreed to buy AveXis for almost $9 billion in 2018, and was soon followed by Roche, which announced in early 2019 it would acquire Spark for $5 billion.
Experts don't foresee the field calming down anytime soon, either. The FDA has said it expects to be reviewing and approving between 10 and 20 cell and gene therapies each year by 2025.
Yet, an influx of advanced medicines could exacerbate some of the existing challenges in making and delivering them. Cell therapy developers like Novartis, for example, have already run into problems commercializing their products, which has caused certain investors to take a more tepid view of the field.
Vineti is hoping its new identifier can iron out some of these problems. As it stands, Vineti's proposal is to have a seven-part ID that would work for all advanced medicine types, including personalized cancer vaccines, cancer immunotherapies, and autologous cell and gene therapies.
The ID would have a "core ID" piece, which shows the company making the product and the specific patient meant to receive it. Then there would be a "treatment ID" piece to identify the product and the order. Lastly, there would be a "specimen ID" piece to detail what procedure is taking place, the run of this procedure, and the number of items produced from it.
So, according to one example posed by Vineti, if a patient is receiving an advanced medicine called ABC from the hypothetical company Acme Pharma, and the first step in that process is to collect one bag of raw material through one session of apheresis, the resulting code would be ACM123456-ABC01-AP0101. In that code, ACM represents the company, 123456 is the patient number, ABC is the therapy, the first 01 is the order number, AP signals apheresis and the last pair of 01s indicate that this is session 1 and should create one bag of material.
Vineti is currently accepting industry-wide feedback on its proposal.