A mother in her early 60s is free of non-Hodgkin lymphoma after participating in a clinical trial for the breakthrough CAR T-cell therapy (chimeric antigen receptor), a customized treatment made from her own white blood cells.
A father in his late 20s is free of sickle cell disease after receiving a novel gene replacement therapy as part of a National Institutes of Health clinical trial.
A young boy can see after receiving a newly approved U.S. gene replacement therapy for retinitis pigmentosa, a rare form of genetic retinal degeneration that leads to blindness.
For patients with difficult-to-treat diseases, such as relentless cancers and inherited genetic disorders, the cell and gene therapy (CGT) industry represents a real beacon of hope. With the growing number of successful product launches, strong financial backing and favorable regulatory environments, EY believes the global CGT market will experience dramatic growth in the next few years. The company predicts revenues will multiply from an estimated US$3b annually in 2021 to an estimated US$50b annually in 2027. With that, the hundreds of CGT patients today will turn into hundreds of thousands by decade’s end.
But is the industry ready?
CGTs have complex manufacturing and supply chain processes that can impact their ability to reach all the people who can benefit. Unlike conventional “1 for many” therapies that are mass produced, CGTs are tailormade “N of 1” treatments requiring the patient to be part of the supply chain. Multiple, flawless handoffs of time-sensitive, unique treatment data and biological materials (human blood or tissue) must occur securely across a diverse set of disparate organizations and information systems, each using their own digital treatment identifiers. While patient outcomes depend on getting these digital handoffs right the first time, they are subject to human error, travel delays, lab closures and even pandemics.
Closing gaps, opening doors for cell and gene therapies
To successfully scale cell and gene therapies, real-time signaling among the players in this extraordinarily complex supply chain will be essential, so that problems can be detected early and speedy course corrections made. All the members of the supply chain – including health care providers, drug companies and manufacturers, lab supply and equipment providers, health services companies and insurers or payers – must be able to securely communicate and share information they trust.
“When you consider all the steps and variables along the cell and gene therapy supply chain and multiply that by the anticipated growth of products and patients, you realize that unless we do something now, there could be a tsunami in store for us,” said Sean Harapko, EY Americas Supply Chain & Operations Solutions Leader.