On May 29, 2019, Be The Match BioTherapies® and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) brought together key stakeholders from the biotech industry, hospitals and apheresis centers, and accrediting bodies for the 2nd Annual Accellerate Summit at our headquarters in Minneapolis, Minn.
Throughout the day attendees discussed opportunities for standardization to streamline access to critical new cell and gene therapies, alleviate confusion and congestion at the point of care, and most importantly help ensure that patients receive safe, high-quality care. Several key themes emerged during the sessions as priority areas for moving efforts forward.
Opportunities to improve the collection process and audits
The desire for standards in collection site audits, training and collections was consistent among all stakeholder groups. Given the growing number of cell and gene therapies in clinical trials today and potential for a large number of commercial approvals on the horizon, these standards need to be developed quickly and collectively to relieve the burden on the apheresis infrastructure.
To create these standards, most participants agreed that cell and gene therapy developers; cell collection sites; regulatory agencies; and groups like Be The Match BioTherapies, CIBMTR, Standards Coordinating Body (SCB), FACT and AABB must collaborate—and soon.
Discussions yielded numerous areas with a critical need for standardization, including:
- Focusing staff training on only the unique aspects of collection for a specific therapy
- Using a standard set of collection specifications
- Limiting the number of audits at cell collection sites, specifically through further development of the Be The Match BioTherapies Quality System Audit Program (QSAP)
- Developing common data collection processes
In order to move these forward, the National Marrow Donor Program®/Be The Match® has already hired additional highly qualified quality and regulatory leaders to support QSAP. This program, originally launched in November 2018, has begun to decrease the number of audits required at apheresis and collection centers, and has proven effective at building efficiencies into the cell and gene therapy supply chain, specifically during the site activation process.
Since the launch of the first iteration of QSAP in 2018, it has become clear that the program can be enhanced further by continuing to gain input from key stakeholders, including industry, accrediting body and apheresis professionals. With a number of near-term approvals on the horizon, minimizing any inefficiencies impacting the collection of starting material, including audit frequency, will be vital to sustainability.
Additionally, Be The Match BioTherapies has hired a consultant to coordinate cell and gene therapy developers; accrediting agencies, including AABB, FACT and the BSMA; and cell collection sites to bring visibility to standardization initiatives and to minimize the potential for duplicative efforts.
Given that eliminating collection process inefficiencies is a top priority among key stakeholders, it is crucial to prioritize the impact that these efforts have on the delivery of life-saving therapies to patients in need.
Standards in the ordering, tracking and delivery of therapies
Another emerging area of concern—particularly for health care professionals—is the number of IT platforms in use for ordering and tracking cell and gene therapies. The number of software systems and the lack of uniform data entry standards currently required of transplant centers directly impacts the speed at which physicians and health care staff are able to provide therapies to patients.
Discussions throughout the day focused on opportunities for industry stakeholders to collaborate in two general directions:
1. The development of uniform data entry standards would better allow systems to integrate and more effectively track the product from ordering through manufacturing and delivery.
2. The development of a single ordering platform for use across the industry could provide health care professionals with the ability to choose the therapy best suited for their patient, regardless of the sponsoring organization or therapy developer. It was additionally proposed that by following therapy selection through the portal, users could be routed to a branded IT experience, which may be critical to some organizations.
As a result of these productive discussions, Be The Match BioTherapies and the CIBMTR have begun exploring the opportunity to provide health care professionals with a single point of entry into the many emerging IT platforms for ordering and tracking the delivery of therapies. Our teams are currently exploring potential sources for funding, both internally and externally, for the development of this type of platform, recognizing that widespread buy-in to the concept of a universal ordering portal must first be achieved. As conversations evolve, both internally and externally, we will continue to engage relevant stakeholders.
Patient outcomes data automation, visibility and sharing
A lack of standardization in outcomes data collection for cell and gene therapies, as well as the current manual entry in reporting databases, both emerged as key concerns in afternoon sessions.
Discussions centered around the potential opportunity to refine the amount of data health care professionals are required to collect and report to track adverse or unanticipated events. Currently, most of the data entry is manual, which leads to inefficiencies when scanning for adverse events, comorbidities, or other clinical information that can influence diagnosis and treatment. By exploring potential for integrations with EMR/EHR systems and other commonly used platforms within hospitals, a higher level of automation and integration with electronic medical records could supply many efficiencies in the context of long-term patient follow-up.
As a result, Be The Match BioTherapies and the CIBMTR are discussing the viability of implementing a more automated process for data acquisition to facilitate the tracking of patient outcomes, as well as developing common data models, to reduce the administrative burden on transplant and cellular therapy programs and help to ensure data integrity across the industry. The organizations are planning to fund the exploration and implementation of electronic reporting as part of a larger initiative to improve accessibility and processes in tracking patient outcomes.
Thank you all for your active participation in the 2nd Annual Accellerate Summit and for your continued commitment to exploring efficiencies in this critical emerging clinical setting. We will provide additional updates as plans are developed and look forward to reporting on progress at the 3rd Annual Accellerate Summit on May 27, 2020 in Minneapolis.