This is no doubt an exciting time in the cell and gene therapy field. Hundreds of cell and gene therapy products are currently in clinical trials. And earlier this year, former FDA Commissioner Scott Gottlieb, MD, and Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, predicted the FDA will approve 10 to 20 cell and gene therapy products every year by 2025.
This rapid growth holds promise for patients, but also challenges for the infrastructure supporting the development and delivery of these therapies. With that in mind, Be The Match BioTherapies® and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) brought together representatives from the biotech industry, hospitals and apheresis centers, and accrediting bodies for the Accellerate Summit in Minneapolis, Minn., on May 29.
Attendees discussed areas of standardization that could be implemented to help ensure that safe, high-quality cell and gene therapies are developed for patients, including:
- Developing standardized approaches in cell collection processes and auditing
- Software platforms for ordering, chain of custody/chain of identity and patient data
- Outcomes data capture and reporting
“Maintaining a focus on patient care, while we identify how to standardize to be more efficient and effective without compromising compliance, is critical. We’re all talking, and that’s a good thing,” said Greg Whitehead, senior vice president, Quality, Rubius Therapeutics, adding, “We all take patient care and patient experience very seriously; it’s what is driving our desire to standardize both the apheresis quality infrastructure and the qualification process. The interest in collaborating for the betterment of patients overrides self-interest for many key stakeholders.”
Attendees agreed the Summit demonstrated the importance of bringing all stakeholders together for open and honest communication about areas where standardization can occur.
“The discussions during the Accellerate Summit were productive and underscored the critical importance of dialogue across all stakeholders in cell and gene therapies,” shared Kara Wacker, MBA, RAC, strategic planning administrator, Foundation for the Accreditation of Cellular Therapy (FACT).
“There is a critical need to harmonize CAR-T cell processing right now. With so much variation in apheresis and cryopreservation methods, and with the accelerated pace of cell therapy clinical trials, we are challenged to harmonize processes and standards. The need is urgent, leadership is critical and stakeholder involvement is requisite,” said Dr. Bob Bowden, head of Cell Collection, Advanced Therapies Supply Chain, Janssen Pharmaceuticals.
Two key challenges emerged as themes throughout the day: the race against time and the number of stakeholders involved, which can make communication and coming to consensus challenging.
“I would say the biggest challenge is that we reach ‘critical mass’ before we land on solutions that work for industry, apheresis and collection centers, and transplant centers. We already see multiple IT systems being implemented in institutions; we see multiple audits with similar scope; we see vast differences in centers with the same accreditations. How long until we reach too many systems to manage, too many audits to conduct, too much disparity that we impact patients? That’s the challenge, beat that clock,” Whitehead shared.
“At the heart of this is open and honest communication and keeping the patient foremost in our minds. We have to continue this conversation and open communication that actively works toward standardization of these processes, and that goes from the beginning of the starting material to the time that it returns. And while we need to do this quickly, we also need to be thorough about it and not just make quick decisions that could have some harmful consequences,” shared Kimberly Kasow, DO, medical director of the University of North Carolina Pediatric Bone Marrow Transplant Program and Marrow Collection Facility and BMTCTP associate director for Quality Initiatives; member, FACT Board of Directors; and vice chair, National Marrow Donor Program®/Be The Match® Board of Directors.
Wacker added, “The cell and gene therapy field is comprised of a wide variety of professions and entities. A big challenge is communicating with and updating each other in regards to the obligations of health care providers, manufacturers, academia, industry, regulatory agencies, standards and accrediting organizations, and payers and then reconciling those obligations into the least burdensome but most effective standards.”
While the conversation at the Summit was meaningful, attendees agree that the work is just beginning.
“It would be a significant accomplishment to gain alignment on apheresis and cryopreservation methods among pharmaceutical/biotech and apheresis/transplant centers in the next 12 months, but I am hopeful. Increased transparency and information sharing is needed because, in many respects, we are already delayed in having these critical conversations. The field is rapidly evolving. To catch-up and ‘get ahead’ of the future of CAR-T will take energy and commitment by all involved to meaningfully impact the current technology and prepare for the future,” Dr. Bowden said.
“This is a really exciting time for the transplant and cell and gene therapy community. But, we need to think through these logistics and how to make it work to be able to be efficient and maintain patient outcomes because that needs to be first and foremost what we keep in our minds. How do we provide these therapies safely to our patients to get the outcomes that we want–which, of course, is our patients alive and well and cured from their underlying disease,” Dr. Kasow shared.
She added, “As a community—and that includes clinical sites, apheresis sites, biotech companies, Be The Match BioTherapies and our accrediting bodies—we all need to communicate, continue to meet as a whole and then really strive and be committed to making changes for the betterment of our patients.”
“FACT is highly committed to our consensus-based Standards development process and to participating in the standardization efforts of related organizations,” Wacker said. “We appreciate Be The Match BioTherapies and CIBMTR for hosting this meeting and are already addressing opportunities that we discovered through conversation with our colleagues.”
“There is a clear appetite amongst all stakeholders to have a forum in which to discuss standards and agreement that standards at the point of cell collection and in IT systems for chain of custody, chain of identity and data capture are needed,” said Chris McClain, vice president, Sales and New Business Development, Be The Match BioTherapies.
He added, “We started standardization work with the industry with the themes that emerged from last year’s Summit and launched the Quality System Audit Program and enhanced the CIBMTR’s outcomes tracking services as a result. We look forward to working with all of the stakeholders in the coming months to continue to move standardization efforts forward at the pace needed to have a positive impact for biotech industry, hospitals and apheresis centers, accrediting bodies and, most importantly, the patients we all serve.”