The Key Force in CGT Commercialization

Since 2020, the world has been in the grips of a faceless and cunning enemy. At this writing, according to the World Health Organization (WHO), there were 233,136,147 confirmed cases of Covid-19, including 4,771,408 deaths.

Thanks to science, we turned a corner with the development of not one, but four vaccines in the US and Europe. And, when we consider that vaccines often take years and sometimes decades to develop, test and approve for public use, we have to believe that something more was in play.

The life sciences industry has increasingly embraced external partnering or “co-opetition” to bring medicines to market faster. But the pandemic has shown how much more is possible with “co-opportunity,” doing the right thing and creating opportunities for all, especially society.

At the height of the crisis, the degree of interorganizational collaboration between private and public entities, as well as large and small organizations, was unprecedented. In an April 9, 2020 press release discussing Pfizer’s partnership with BioNTech on a Covid vaccine, Mikael Dolsten, Pfizer chief scientific officer and president, worldwide research, development and medical, discussed how combating the pandemic will require unprecedented collaboration across the innovation ecosystem, with life sciences organizations coming together to unite capabilities like never before.

Pharmaceutical and biotechnology companies, academia and technology joined forces to deliver rapid vaccines, diagnostic tools and therapeutics. It was, in effect, an industrialized level of collaboration that, if replicated in the cell and gene therapy (CGT) industry, would be the single most important factor in scaling these next-generation therapies.

Why and how should the CGT industry build on the momentum of pandemic partnerships to create a collaborative culture and an interoperable, digital ecosystem that ensures scalability?

The industrialized collaboration movement

Medicine is transitioning—from mass-produced drugs to individually tailored therapies, from maintenance regimens to one-time curative treatments. With 17 approved CGTs to date and an active pipeline of reportedly more than 3,000 treatments in various stages of development, there could be an influx of 200 to 300 CGT product approvals between 2022 and 2027.

But these drugs involve complex manufacturing processes and a complicated, onerous supply chain. Currently, patients can wait an average of six to eight weeks for their treatments, and up to 90% of CGTs are not delivered as originally planned. In addition, advanced therapies come with higher-than-average prices.

How can these life-changing therapies scale up to help all those who can benefit, when the thousands of CGT patients today turn into hundreds of thousands by decade’s end?

It’s going to take a movement, called “industrialized collaboration,” to advance CGTs. Building on the momentum of collaboration inspired by the coronavirus pandemic, its purpose is to help ensure CGT universal accessibility. And its vision is one of an interoperable CGT ecosystem.

The barriers to universal CGT accessibility

The journey of an individual CGT, from the patient to the clinician, to apheresis collection, to multifaceted manufacturing and back to the patient, presents three main challenges:

1. Fragmented supply chain. Instead of the well-worn one-directional supply chain of mass-produced therapies, autologous CGTs depend on a bidirectional supply chain with nearly triple the number of steps. CAR-T therapy, for example, involves over 40 coordinated digital and analog handoffs between disparate teams of healthcare providers, manufacturers, third-party logistics companies (3PLs) and payers. Maintaining chain of identity and chain of custody while reducing the time it takes for therapies to move from “vein-to-vein” is critical. Some allogenic CGTs also have complexities in their supply chains that must be overcome. For both, the supply chain is fragmented, with many different software systems that don’t “talk” to each other, limiting members’ real-time visibility along the patient and therapy journey.
2. Bespoke manufacturing. With the expected increase in patients, the industry is focused on solutions that scale at 1,000 times the current rate. Companies are investing in technologies and larger production facilities; creating processes to help products scale or creating new products that scale better; and using technology to streamline quality control without sacrificing integrity or compliance. Still, the timelines are often too long and the therapies too expensive to reach many patients.
3. High prices. While CGTs can deliver tremendous value, there is uncertainty around response durability. Current models of drug pricing, which are based on utilization, won’t work well. CGT companies and payers are considering new payment models, including outcomes- or value-based contracts (OBCs/VBCs), “drug mortgages” and subscription-based models, which charge a flat rate. Furthermore, there is an urgent need, highlighted by the pandemic, to extend essential services and offerings to the disadvantaged.

The vision: an interoperable CGT ecosystem

The Covid-19 pandemic was declared by WHO to be a global emergency, a fire that needed extinguishing. But there are millions of people around the world battling their own fires, such as cancers, inherited genetic disorders, autoimmune diseases and chronic illnesses such as heart disease, diabetes, hemophilia, and HIV/AIDS. The CGT revolution offers them hope for a better, longer life and maybe even a cure.

Many in the industry are calling for better collaboration and industry standards—including a unified digital platform—to cost-effectively and safely facilitate the anticipated high volume of therapies and patients. But how do you build a plane while you are flying it?

Cultivating a culture of industrialized collaboration

At the Alliance for Regenerative Medicine’s Meeting on the Mesa in October, we asked participants to a workshop we hosted: What will be the greatest barrier preventing the needed collaboration to scale the delivery of CGTs—now and in five years? Respondents said organization and industry culture (23% now and 25% in five years) along with treatment journey complexity (19% now and 25% in five years) was at the top of the immediate and forward-looking time frames. We need to focus our attention on solving both.

Not until Covid have we witnessed the level of partnership required to address the CGT industry’s current shortcomings. It is on an industrial level, where collaboration is woven into and throughout the fabric of the CGT industry and input is sought from all relevant stakeholders. These include the life sciences industry, logistics firms and medical, regulatory, policy, payer and patient representatives.

Transforming supply chain into value chain

Now that patient numbers are small, the supply chain relies on highly manual processes by many skilled operators. But as therapies, patients and human touchpoints multiply, so will variability and risk. There is a need for a digital infrastructure that is secure, trusted and interoperable, to facilitate collaboration and real-time transparency and to ensure future scalability, shorter treatment delivery times and reduced costs.

EY teams estimate that a shared digital infrastructure can save the industry roughly $1 billion that they would spend building their own data systems. EY believes that a unified platform can also inform and improve treatments today and tomorrow.

About the Author

Adlai Goldberg is EY’s Global Digital, Social and Commercial Innovation Life Sciences Leader.

The views reflected in this article are the views of the author and do not necessarily reflect the views of the global EY organization or its member firms.