News Hub

by

Kara Wacker ,MBA, RAC

Strategic Planning Administrator

Foundation for the Accreditation of Cellular Therapy (FACT)

"Great things in business are never done by one person; they're done by a team of people." —Steve Jobs

FACT’s mission is to improve quality cellular therapy for the benefit of patients. Adapting to rapid advances in science and paradigm-shifting methods of delivering health care is what we do, and we have been successful thanks to the input of a variety of perspectives.

FACT has collaborated with multistakeholder groups since it was founded in 1995. Our very genesis was the result of collaboration between the American Society for Transplantation and Cellular Therapy (ASTCT) and the International Society for Cell & Gene Therapy (ISCT) to develop the first comprehensive set of Standards for hematopoietic progenitor cell transplantation.[i] Since then, FACT has worked with many organizations. In 1999, we co-published the Hematopoietic Cell Standards with JACIE to expand quality beyond U.S. borders. In 2000, we partnered with NetCord, now part of WMDA, to create standards and accreditation for cord blood banking.[ii] Since 2013, we have served as a recognized accreditation for the United States National Cord Blood Inventory (NCBI)[iii] and have conducted supplementary inspections against NCBI-specific criteria. We also partner with organizations on quality initiatives, such as the joint Data Audit Committee with the Center for International Blood & Marrow Transplant Research (CIBMTR).[iv] Other stakeholders we work with include ICCBBA, the American Society for Apheresis (ASFA), the Cord Blood Association (CBA), Be the Match, and more.

The Advent of Commercially Manufactured Cellular Therapies and the Need for Multistakeholder Cooperation

Following successful clinical trials of chimeric antigen receptor (CAR) T cell therapies, FACT published its Standards for Immune Effector Cells[v] in 2017 and implemented an associated accreditation program at the request of accredited programs, FACT inspectors, and payers. The commercialization of the first licensed CAR T cell products[vi] that year led FACT to expand our circle of collaborators to commercial and clinical trial product manufacturers.

The transition from clinical trials to commercial manufacturing led to unexpected challenges for health care institutions and manufacturers alike. One of these is duplicative audits by multiple manufacturers at FACT-accredited sites that already undergo rigorous inspections on a regular basis. For their part, manufacturers were trying to implement processes in compliance with their regulatory submissions within disparate health care sites using collections from inherently variable human donors for cellular starting material.

There was a strong desire from stakeholders to leverage FACT accreditation to identify sites that could collect cellular starting material and administer final cellular therapy products within the context of Good Tissue Practices (GTPs) and quality systems. FACT-accredited sites wished to minimize the redundancy of manufacturer audits, and the conflicting observations that result.

FACT’s first interactions with our manufacturer colleagues consisted of telephone calls, emails, and in-person introductions. Through comparison of FACT Standards to manufacturers’ audit checklists, review of manufacturers’ audit observations, and communication on challenging issues between industry and health care sites, we identified ways to clarify or add to our Standards to maintain the relevance of FACT requirements amid the exciting evolution of cellular therapies. We also demonstrated that our robust accreditation program can accommodate the transition from clinical research to commercialized therapies at sites while fulfilling regulatory obligations of manufacturers.

These activities were insightful, but done in silos one manufacturer at a time. Complex challenges require a wide variety of perspectives representing the entire value stream of cellular therapy, including starting material collection, laboratory processing and storage, chains of identity and custody, distribution, administration, and patient follow-up. Eventually, many organizations created multistakeholder groups to address these challenges.

FACT Participation in Multistakeholder Groups

Multistakeholder groups have generated ideas and practical solutions that have the potential to reduce bottlenecks in cellular therapies and thereby increase patient access. Though listing all multistakeholder groups is beyond the scope of this article, the following are examples of groups in which FACT participates to address redundancies:

·         ASTCT 80/20 Task Force: This task force considers challenges and potential solutions to issues related to duplicative documentation requirements for each approved cellular therapy product and proposes that 80 percent of manufacturers' requirements for onboarding and ongoing operations of commercially available products could be standardized and streamlined.[vii]

·         Deloitte NextGen Industry Working Group: This working group provides a platform for cell and gene therapy leaders to exchange perspectives, share best practices, and develop solutions, including streamlined and more rapid site certification.[viii]

·         Standards Coordinating Body: The Standards Coordinating Body (SCB) complements current Standards Development Organizations and their processes for standards development by engaging regenerative medicine stakeholders.[ix]

FACT Implementation of Multistakeholder Solutions to Reduce Duplicative Assessments and Audits

Via our work with multistakeholder groups, we confirmed that a majority of manufacturers’ needs for assessing collection and treatment sites are already covered by compliance with FACT Standards. Manufacturers need documented assessment of sites that specifically verifies compliance with the applicable regulations as they apply to their cellular therapy products. The following are ways in which FACT is addressing manufacturers’ needs to reduce burden on health care institutions:

·         Soliciting manufacturer input in the FACT Standards development process: FACT Standards are developed by consensus of experts, based upon published medical evidence and best practices. By understanding manufacturers’ needs and regulatory framework, standardized requirements can be developed to advance the field. By proposing and vetting those requirements via the FACT Standards Development process, incorporating them into draft consensus standards, and reviewing public feedback, the result is increased standardization. This can reduce auditing obligations, simplify quality agreements, and direct more resources to product manufacturing and patient care.

·         Supporting manufacturers’ efforts to leverage FACT accreditation: FACT provides manufacturers documentation of how its Standards, inspections, and annual reports relate to regulations. We also provide policies, procedures, regulatory crosswalks, and other documents to support manufacturers’ due diligence processes. Finally, though not required, we acknowledge to accredited programs the benefits of providing their final accreditation report to manufacturers when requested. These activities support progress toward a single assessment that is already recognized by several stakeholders.

·         Advocating for the implementation of multistakeholder solutions: Examples of multistakeholder solutions implemented by organizations include ISBT 128 standards for labeling of cells collected for further manufacturer and a Chain of Identity (CoI) Identifier published by ICCBBA, the Cellular Immunotherapy Data Resource (CIDR) data registry managed by CIBMTR, and the Risk Assessment and Scope Determination Forms developed by the Deloitte NextGen IWG Site Certification Workstream. These solutions offer streamlined processes that are in compliance or harmonized with FACT Standards and accreditation.

·         Expanding FACT services to manufacturer-requested site assessments: To support adoption of rapid developments in cellular therapies and high patient demand, FACT will conduct cellular therapy product-specific assessments beyond the current FACT accreditation program. This includes product-specific surveillance audits in conjunction with FACT inspection visits, provision of qualitative data regarding accredited sites to manufacturers as part of their site qualification processes, and evaluation of non-FACT-accredited sites, providing assistance in meeting GTP regulations.

Building FACT Assessment & Audit Services

The two main goals of FACT’s assessment and audit services will be to reduce redundant assessments at FACT-accredited sites already qualified to participate in cell therapy and more quickly and effectively qualify additional sites.

We believe that FACT is a natural fit for assessment and audit services because we can:

1.      Review a larger sampling of records representing more than an individual manufacturer, leading to better identification of trends and potential systemic noncompliance.

2.      Standardize assessments while reviewing product- or protocol-specific items as needed.

3.      Guide sites on how to correct deficiencies, potentially shortening the time to closure of the assessment.

4.      Coordinate and streamline assessments with its accreditation process, further reducing duplication and potentially reducing the need for in-depth surveillance audits.

FACT hosted an industry focus group meeting during Advanced Therapies Week in January to present our preliminary plans and gather feedback from manufacturers and other stakeholders. We are using this feedback to develop a menu of services, a core assessment and audit checklist, proposed deliverables, and a pricing structure. These items will be provided to an industry advisory group for further refinement. This year, we plan to conduct pilot assessments and audits with participating companies to verify and further develop our processes.

If you are interested in participating in the development of FACT assessment and audit services, or utilizing our services, please contact Liz Ferraro, Manager of Assessment and Audit Services, at liz.ferraro@factglobal.org. We plan to incorporate as many perspectives as possible. Our intention is to develop these services to be a WIN-WIN-WIN for health care institutions, manufacturers, and, most of all, patients. 

#RegionalandPartnerUpdates