Adlai Goldberg, EY 

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 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

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