Adlai Goldberg, EY 

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:

 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.

 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.

 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.

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.

Articles

Industrialized collaboration is critical to taking next-generation cell and gene therapies to the next level—if companies can build on pandemic-partnering momentum

The Key Force in CGT Commercialization

The world is reaping the benefits of more than 40 years of continuous innovation in the cell and gene therapy (CGT) field. We are witnessing transformative and even curative treatments for difficult diseases such as inherited disorders and cancers.

The goal of this article is to help key players in the value chain think strategically about what it will take to scale up the CGT industry to the point where it can deliver many more therapies to many more people, so that these treatments of last resort become therapies of first choice. EY expects the CGT global market to grow exponentially faster than the total pharmaceutical global market, from an estimated $3 billion annually in 2021 to more than $50 billion yearly by 2027. The more than 2,600 CGT clinical trials in different modalities at different stages of maturity signal a potential wave of product approvals between 2022 and 2027.

As Orlando Serani, program lead of advanced cellular therapies supply chain at Johnson & Johnson, said at the 

, “… cell and gene therapy science is delivering therapies faster than the digital environment can support them. While we must play catch up, we don’t know for sure what we’ll need in two to three years’ time. We need to build the interoperable foundation and adapt it from there.”

As a result, it is expected that the volume of patients treated with CGTs will dramatically rise from the hundreds today, to the tens of thousands by decade’s end.

CGTs represent a departure from pharmaceuticals, in that their manufacturing and delivery consist of a complex, often varied process with zero margin for error, shifting the treatment paradigm from a mass market (1-for-many) to an individualized approach (N of 1). The Covid-19 pandemic tested the limits of the current ecosystem, underscoring vulnerabilities in manufacturing and delivery, research and development and commercialization.

One essential problem is that CGT supply chains require the seamless orchestration of time-sensitive materials and information across a diverse set of organizations and systems. Today, this process is largely managed by people and is therefore subject to human error, as well as travel delays, lab closures, etc. According to Sean Harapko, EY Americas supply chain and operations solution leader, “… to successfully scale cell and gene therapies, real-time tracking and tracing across the full value chain is essential, so that problems could be detected early and speedy course corrections made.”

The second big hindrance to making CGT a reality at scale is cost. Highly specific CGTs mean complex manufacturing, short-term treatment and curative-intended outcomes, which mean higher-than-average prices. But their treatment duration remains unknown and current models of drug pricing, which are based on utilization, won’t work. The result—currently limited availability, to a small number of patients. Several innovative payment and risk-sharing approaches are being developed and considered to address this issue. These include 

 (OBCs), which tie a product’s performance to emerging evidence of improved patient outcomes and “drug mortgages” that would allow patients and payers to schedule payments of a value-based cost over a fixed period of time. Stakeholders remain concerned that OBCs are too risky, too complex to design and measure and too difficult to replicate across multiple parties. But through 

, EY has discovered that leveraging new digital technologies that make it easier and safer to collect and share data will eliminate some of the long-standing frictions associated with OBCs.

The path forward: Collaboration, data and trust

The Covid-19 pandemic also revealed the necessity for unprecedented sharing of information to drive the collaboration and coordination required between patients, hospitals, logistics companies and biopharma companies. As Serani emphasized at the event, “… we need collaboration at the speed of science.” Every process and hand-off in between need to be seamless and precise, as individual biological materials travel from patient to provider, to transport company to biopharma manufacturing site, and back again. Mistakes could result in dire consequences or even death.

Pamela Spence, EY global health sciences and wellness leader, believes data is the key here: “The global disruption caused by the Covid-19 pandemic has demonstrated that resiliency, agility and innovation are more possible with digitally enabled business models that have data at their core.”

In CGT, that translates into a digital ecosystem that is built on trust and partnership; one that supports data collection, management and sharing; evolves and expands to manage rapid growth; and becomes the foundation for delivering value.

According to the World Health Organization, 

 a year will be diagnosed with cancer or other mutation-based diseases. What could the future of CGT look like? Shared information, trust and an unprecedented level of collaboration among the key players in the CGT ecosystem will mean faster, more efficient and accessible treatments. These attributes will also help bring about dramatically increased success rates, expanded offerings and lower costs.

This recipe for resiliency requires the foresight to put the right systems in place that will serve patients and the industry now and into the future.

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

The importance of thinking strategically about the infrastructure and foresight needed to scale up CGTs—not as treatments of last resort, but first choice