The FDA’s approval in December of a new gene editing treatment for sickle cell disease not only represents a potential cure for a painful illness that afflicts millions of people globally but could be a harbinger of a surge in cell and gene therapy (CGT) activity throughout 2024 and beyond. This prediction is supported by a report from the American Society of Gene and Cell Therapy1 that as of the third quarter of 2023, there were 3,866 therapies in various stages of development. McKinsey has projected2 that there will be 22 cell therapy and as many as 31 gene therapy launches in 2024.
As promising as all that activity is for public health and CGT market players, it exposes a potentially serious issue with patient and provider access—one that threatens to complicate providers’ work, delay patient access to treatments, and hinder overall market development. This threat isn’t from the therapies themselves, but rather from the lack of standardization across the platforms that patients and providers use for onboarding and coordinating patient treatment.
Today there seem to be as many access portals as there are commercially available CGT treatments, with each portal having its own unique processes and requirements. If cell and gene therapies are going to be fully scalable and fulfill their market potential—Vantage Market Research projects the global CGT market will grow at an average annual rate of 18.2% this decade3—then the focus must be on delivering a standardized user experience via an integrated hub that providers and patients can easily navigate to access and manage delivery of multiple treatments from multiple manufacturers.
As the CGT market grows, so will the demands upon treatment centers and their staff to coordinate patient onboarding and treatments across multiple portals, which will be labor intensive and, potentially, prone to errors and cybersecurity threats.
No single player can solve this problem alone. It’s an industry-wide issue that will require an unprecedented level of collaboration and consensus among manufacturers and other members of the CGT ecosystem, including treatment facilities, ingredient suppliers, contract manufacturers, logistics providers, laboratories, and insurers.
A recommendation
Our recommendation is that entities across the CGT value chain make a concerted effort to create a standardized environment where providers can access a full menu of treatments from multiple manufacturers, then manage all the processes around the patient journey, from onboarding to ordering, scheduling, and through to administration of the therapy, and do so in a digital environment that’s intuitive, secure, transparent, and compliant. Delivering all this on the front end will require a high level of coordination, collaboration, trust, and transparency on the back end, with full visibility into both the patient and product journeys across an array of stakeholders. That is because, ultimately, with cell and gene therapies, the product itself is only as viable and effective as the processes behind it.
The starting point for this effort is to embrace co-innovation, where manufacturers and other members of the CGT value chain commit to collaborating to develop a shared hub, recognizing that it is in the best interests of patients, providers, their own companies, and the overall CGT market. Manufacturers, in particular, must see the business case for shifting away from single-tenant portals and instead sharing costs and risks with other manufacturers.
Ultimately, this will enable them to differentiate themselves based on the quality of their therapies, not the quality of their portal. For a universal hub to become truly global, this mindset will also need to extend across jurisdictions, markets, and borders, and involve international organizations and regulatory agencies as well as the private sector.
For such an ambitious effort to succeed, entities across the CGT value chain must mobilize to ensure several foundational back-end elements are in place:
- Connected business networksin which all parts of the value chain—from the patient, to their treatment provider, and all the way to manufacturers and their suppliers and contractors—are sharing data in real time about the production and movement of critical materials and specific patient treatments, with full visibility into changing circumstances related to the patient, suppliers, etc., so that potential disruptions can be readily identified, alerts issued, and adjustments made accordingly. The concept here is to create a digital thread or chain of identity/chain of custody to provide visibility into all the key data associated with a particular therapy and its journey to the patient.
- Trust among those sharing data within these networksthat data will be secure, and that they can maintain provenance over competitively sensitive information.
- Widely accepted standards and policiesthat apply to front- and back-end systems and processes, the handling of electronic medical records, secure data management and exchange, communications among members of the value chain, and more.
- Modern, intelligent digital systemsthat enable manufacturers, logistics providers, and other links in the supply chain to contribute to the digital thread, with the ability to pinpoint and share fresh, accurate data relevant to their role in the product journey. For some companies, this will mean shifting away from manual-intensive, siloed processes and systems.
- Real-time communications channels across the CGT value chain, so that parties can follow the digital thread and adjust to cascading changes that impact manufacturing, logistics, scheduling, and—ultimately—the patient, whether it’s an issue with an ingredient supplier that could delay production, or an unexpected development with a patient that could delay treatment.
- A cloud-based platform architecturethat would provide maximum accessibility,flexibility, and scalability.
How can we coordinate all of these moving parts to make this vision a reality? The good news is that we’re not starting from Ground Zero. Groups such as the Cell and Gene Therapy Consortium4 and BioPhorum5 are already laying the groundwork. And individual companies have begun embracing a co-innovation mindset, in the hope that their peers will join them.
Strengthening and streamlining back-end processes to create a universal hub for CGT will go a long way toward establishing a sustainable business model where newly commercialized therapies reach people in a safe and timely way, and where all the various parts of the CGT value chain prosper in whatever role they play in getting treatments to patients in need.
At SAP SE, Mazahir Valikarimwala is solution manager, life sciences, for cell and gene therapy orchestration; Eduard Hergenreider, PhD, serves as a product manager, life sciences, cell and gene therapy orchestration; and Martin März PhD, is also a product manager, life sciences, cell and gene therapy orchestration.
References
1. Armstrong SE, Mariano JA, Lundin DJ. (2010) The scope of mycoplasma contamination within the biopharmaceutical industry. Biologicals 38, 211–213.
2. U.S. Government (2010) Code of Federal Regulations, Title 21, Subpart D-Mycoplasma §610.30.
3. U.S. Government (2010) Code of Federal Regulations, Title 21 §610.18(c): Cell lines used for manufacturing biological products.
4. United States Pharmacopeia (2010) <63> Mycoplasma Tests.
5. European Pharmacopoeia (2008) Chapter 2.6.7 Mycoplasmas.
