In its latest snapshot of the cell and gene therapy market, the Alliance for Regenerative Medicine declared a “record year” for treatment approval in a “a landscape of steady progress, notable achievements, and resilience.” As of the third quarter of 2022, more than 3,600 therapies were in development, according to a recent report from the American Society of Gene & Cell Therapy (ASGCT) and Citeline. More gene and RNA therapies were approved in the past quarter (five) than in each of the previous quarters for more than a year. And McKinsey projects another 21 cell therapy launches and as many as 31 gene therapy launches in 2024 alone.
For patients and pharma manufacturers alike, the motivation and the need are there. Manufacturers are aggressively seeking ways to produce cell and gene therapies faster and less expensively to meet growing consumer demand for personalized treatments for cancers and rare diseases. Meanwhile, the American Chemical Society cites projected annual sales growth of about 15% for cell therapies and close to 30% for gene therapies.
Yet the vast potential of the cell and gene therapies market remains largely untapped as 2023 approaches — the result of longstanding fundamental hurdles related to development cost and time, quality assurance and approval timelines, reimbursement structures and supply chain risk, to name several. What’s lacking is a clear, sustainable way to scale production and ensure supply chain integrity so that treatments are profitable for manufacturers and other segments of the value chain, and safe, timely, effective and more affordable for patients. To date, relatively few treatments have been approved and those that have been remain cost-prohibitive and/or inaccessible for many potential patients. Globally, as of Q32022, 21 gene therapies and 22 RNA therapies had been approved for clinical use, along with 59 non-genetically modified cell therapies, according to the ASGCT.
As formidable as the obstacles to building a more robust market for cell and gene therapies may seem, they are not insurmountable. The key to unlocking the market’s potential could well be co-innovation: stakeholders across the industry coming together to develop and implement solutions, and in doing so, accomplish what no single stakeholder could do on its own. Here are five areas where co-innovation could help cell and gene therapies and other personalized treatments fulfill their massive potential:
1. Creating connected business networks
Bringing cell and gene therapies to market already requires coordination among a diverse array of stakeholders, from manufacturers and contract manufacturers to laboratories to logistics companies to treatment providers. What’s needed now is to further elevate cooperation by establishing digitally connected ecosystems within which stakeholders share data and insight as part of business network, with their interests aligned around developing and bringing to market a larger volume of treatments across a broader range of modalities.
Making this a reality requires a shift in mindset, where manufacturers (some of whom may be more accustomed to competing against one another than collaborating) agree to share risk, reward and information across a shared platform. For some companies, it also will entail a modernization and integration of digital capabilities, and a move away from manual-intensive, siloed processes and systems, so they can readily access and share trusted data in real-time across the business network.
Through these ecosystems, stakeholders also could collaboratively develop open business-process Application Programming Interfaces (APIs) that are interoperable across manufacturing platforms and organizations. These APIs could become the catalyst for event-driven data exchange, open communications, visibility and transparency across such an ecosystem.
2. Reimagining the supply chain
The unique demands involved in getting cell and gene therapies to market require a progressive approach to supply chain, one that ensures coordination from the manufacturer, through labs and logistics providers, to treatment facilities, payors, all the way to the patients themselves.
The vision is clear: ensure the right treatment reaches the right patient at the right time, with zero tolerance for errors. Achieving that vision is a major challenge, due largely to a lack of visibility among stakeholders across the supply chain that can hamper efforts to track transportation of treatments, and ensure product integrity and patient safety. A ‘digital thread’ or chain of identity/chain of custody would bring much needed visibility into all the key data associated with a particular therapy and its journey to the patient. Armed with real-time data from the digital thread, parties can synch up and make better-informed supply chain decisions every step of the way, cutting to-market cycle times significantly.
3. Streamlining quality assurance for individualized products
As lengthy as the approval process for cell and gene therapies tends to be, integrating more automated and exception-based processes into quality assurance could speed QA and approval processes significantly, while reducing the risk associated with manually intensive, more error-prone processes. Here’s another area ripe for co-innovation, where stakeholders could collaboratively develop and apply standard, automated QA processes so manufacturers can shift to a faster and less error-prone review-by-exception approach. They could collectively implement a variety of automated checkpoints into the process to ensure adherence to a set of common practices, processes, systems and functions, which in turn could lead to faster development and approvals.
4. Standardizing a treatment center platform to provide access to multi-vendor offerings
Organizations offering cell and gene therapies could really benefit from a time-sensitive, fault-tolerant orchestration engine — a centralized hub — to manage the flow of samples from treatment centers to manufacturing locations and then the return of the personalized therapies for administration at treatment centers. Patients and manufacturers also would benefit from a shift away from single-tenant portals, to a standardized platform or hub where patients can access therapies from multiple providers. This could be a real catalyst for rapid scaling. Collaboration among stakeholders can make these kinds of innovations a reality.
5. Developing new reimbursement concepts
The uniquely individual nature of cell and gene therapies demands new, broadly accepted reimbursement and pricing models, such as outcome-based reimbursements. We expect such a model would be widely embraced in light of the high cost of treatments, as it is a value-based approach that spreads risk among all stakeholders.
The impetus for stakeholders along the cell and gene therapy value chain to come together and co-innovate is indeed strong on both the supply and demand sides. And today, we’re seeing some of the world’s largest manufacturers take steps toward developing supply chain-focused business networks (with supporting cloud-based digital platforms) to make the aforementioned improvements a reality. With these networks in place, expect to see more record-setting years where approved treatments number in the dozens or perhaps even in the hundreds annually and they’re widely available to patients who need them, at the right time and at a significantly lower cost.