On May 30, Be The Match BioTherapies® brought together key cell and gene therapy professionals to discuss two big challenges that are impacting the entire industry: system capacity and the need for standardization. The main goal of this event? Brainstorm, collaborate and strategize ways to develop standards that will help to ease the current burden on apheresis centers and transplant centers, which are so integral in the infrastructure for the successful delivery of cell and gene therapies.
Hosted at our headquarters in Minneapolis, the first Be The Match BioTherapies Cell and Gene Therapy Standardization Summit sparked cross-functional discussion and sharing of perspectives between all attendees, including companies developing and commercializing cell and gene therapies, like Kiadis Pharma, Orchard Therapeutics and Atara Biotherapeutics; apheresis and transplant center physicians, nurses and administrators; payer and reimbursement professionals; members of accrediting and regulatory bodies; and other biopharma leaders.
As Amy Ronneberg, President of Be The Match BioTherapies, put it in her opening remarks, “Although the next five years provide more opportunities than we’ve seen in a long time in the industry, that also brings challenges.”
Chief among those concerns? System capacity and the increasing number of audits and training being requested at organizations collecting cells and delivering therapies—often with overlapping requirements.
Heather Steinmetz, MPH, is the Quality Assurance Manager in the Hematologic Malignancy/Stem Cell Transplant Program at UCLA Health. She provided some insight into the audit and training redundancies.
For example, between October 2017 and April 2018, the organization went through a total of four audits, which included not just the expected regulatory audits, but also individual commercial audits. It also experienced six site visits, with four of these being onsite audits, of the Hemapheresis Unit for clinical trials, with the majority related to CAR-T/engineered cell therapies.
“Because some audits are nearly back to back, CAPAs may still be in progress when the next audit occurs, potentially leading to identical findings in multiple reports with varying levels of CAPA acceptance from companies,” she said.
Additionally, UCLA’s stem cell lab staff has been repeatedly trained by multiple suppliers on how to open a dry shipper and put cells in a storage tank. “We have a standard operating procedure in place which includes staff competencies that is reviewed at each audit. Yet, each supplier wants its own training and its own audits as well. Such duplication creates delays in administering the treatments that patients need,” Steinmetz said.
“The industry needs to be doing more to support apheresis centers and others at the heart of this infrastructure,” said Amy Hines, Be The Match BioTherapies’ Director of Collection Network Management, and a former Transplant Nurse. “We’re continually adding to our Be The Match BioTherapies collection network support team and expanding our quality and regulatory services team. Our goal is to provide universal audits that are accepted by cell and gene therapy companies, while also ensuring that centers are fully compliant with individual therapy needs. This would allow an apheresis center to avoid multiple audits for nearly identical requests and minimize the burden on apheresis staff.”
The attendees flagged other important concerns as well, including how to produce consistent quality starting material for new therapies, payment and reimbursement issues, developing consistent industry best practices, and creating an action plan for industry-wide standardization. In small groups, they brainstormed solutions for each of these challenges, and strategies for implementing them.
“To me, this is truly the most exciting thing I have ever seen in medicine. We are at the beginning of this and I think we have the opportunity to shape this really well and really quickly,” said David Porter, M.D., Director, Cell Therapy and Transplantation at the University of Pennsylvania and Abramson Cancer Center
As a next step, Be The Match BioTherapies will be developing a working committee to bring the conversation forward to other key industry stakeholders and begin to implement an action plan for industry-wide standardization.
If there is one thing that everyone in attendance agreed on, it was the great potential for cell therapies to transform the future of how diseases are treated, and ultimately improve care for patients.
Because, as Amy Ronneberg remarked, “At the end of the day, we are all here for the patients.”
Onboard cell collection facilities faster by obtaining our audit results measuring compliance with Good Tissue Practice (GTP), Good Manufacturing Practice (GMP)
and other applicable quality measures.