The 20th year of FACT successfully built upon its history of successes to advance cellular therapy and regenerative medicine. As physicians and scientists continue to make advancements, opportunities arise for FACT to play a supporting role to educate all programs on new approaches and therapies. The following are highlights from 2016:
1. FACT Celebrates 20th Anniversary
FACT celebrated its 20th year as the leading organization in standards setting and accreditation for cellular therapy, hematopoietic stem cell transplantation, and regenerative medicine. FACT welcomed visitors to a dynamic exhibit booth at the BMT Tandem Meetings and the ISCT Annual Meeting to celebrate with the organization. Attendees who wore their commemorative FACT 20th anniversary pin received gifts and were entered in a daily drawing for prizes.
FACT also hosted anniversary receptions at these meetings, and many FACT colleagues, volunteers, and friends enjoyed hors d’oeuvres and refreshments as FACT celebrated two decades of working together in the community to improve patient care and safety. Guests enjoyed watching a short video highlighting FACT’s past 20 years, and saw some familiar faces who have been an integral part of FACT since its beginning.
2. FACT Commences Review of Corrective Action Plans for Low One-Year Survival
The FACT Clinical Outcomes Improvement Committee began formally reviewing corrective action plans submitted by Clinical Programs that did not meet expected one-year survival as outlined by comparative data. The emphasis of the committee is to help programs identify ways to improve their outcomes for patients. After several months of reviewing corrective action plans, the committee has articulated its expectations for these plans using six guidelines:
- The corrective action plan must identify specific causes of death.
- The corrective action plan must provide quantitative data.
- The assessment must identify reasonable causes of the low one-year survival rate.
- The corrective action plan must address the identified causes.
- There must be a measurable outcome improvement.
- The program must provide updates on corrective actions at the time of inspection, at the time of annual reporting, or as otherwise directed by the committee.
3. FACT Presents Comments at FDA Hearing
The Food and Drug Administration (FDA) hearing to obtain comments on the four draft guidance documents relating to the regulation of human cells, tissues, and cellular and tissue-based products (HCT/Ps) occurred on September 12-13, 2016. Dr. Phyllis Warkentin spoke on behalf of FACT and presented the following comments to the FDA:
- FDA should fulfill its responsibilities to protect patients in search of cellular therapies. FACT supports its parent society, the International Society for Cellular Therapy (ISCT), in its position on unproven therapies and agrees on the importance of providing adequate education for patients. Development of professional standards and voluntary accreditation can play an important role in providing a bridge from basic research to clinical application.
- The tiered unified approach to HCT/P regulation fails to acknowledge the complexity of some tissues with multiple native functions and many cell types. It is difficult to strictly categorize complex tissues such as adipose tissue as only structural or cellular. Dr. Warkentin offered solutions for applying regulations to such tissues.
- There appear to be some inconsistencies in the definitions and examples of homologous use that would benefit from clarification.
- FACT suggests that the Agency expand upon its expectation for cord tissue, to include which regulations apply and when they apply, based upon the processes in place.
- International harmonization is important to facilitate product development and world-wide availability of cell-based therapies for patients.
4. Sixth Edition NetCord-FACT Cord Blood Standards Published
NetCord and FACT published the 6th edition NetCord-FACT International Standards for Cord Blood Collection, Banking, and Release for Administration and its accompanying Accreditation Manual on July 1, 2016. These Standards became effective after 90 days, and all accredited cord blood banks were required to comply with the requirements by that time.
These Standards cover 1) collection of cord blood cells, regardless of the methodology or site of collection; 2) screening, testing, and eligibility determination of the maternal and infant donor according to Applicable Law; 3) all phases of processing, cryopreservation, and storage, including quarantine, testing, and characterization of the cord blood unit; 4) making the cord blood unit available for administration, either directly or through listing with a search registry; 5) the search process for selection of specific cord blood units; 6) reservation and release of cord blood units for clinical use; and 7) all transport or shipment of cord blood units, whether fresh or cryopreserved.
5. Draft 1st Edition Immune Effector Cell Standards Published for Public Comment
FACT published a draft of the 1st edition FACT Standards for Immune Effector Cells for inspection and public comment in July 2016. Interim standards for the 6th edition FACT-JACIE International Standards for Hematopoietic Cellular Therapy Product Collection, Processing, and Administration were also proposed to include immune effector cell programs within blood and marrow transplant units. These Standards are intended to promote quality in administration of immune effector cells and will be incorporated into a voluntary FACT accreditation in this field.
The Standards apply to immune effector cells used to modulate an immune response for therapeutic intent, such as dendritic cells, natural killer cells, T cells, and B cells. This includes chimeric antigen receptor T cells (CAR-T cells) and therapeutic vaccines.
Commenters included, among others, pharmaceutical companies, the FDA, the American Society of Cell & Gene Therapy (ASGCT), and the Society for Immunotherapy of Cancer (SITC). A total of 131 comments were received, and reviewers generally expressed support for the Standards and posed worthy questions to consider.
6. CYCORD is First Cord Blood Bank in Cyprus to Earn FACT Accreditation
CYCORD Public Allogeneic Cord Blood Bank in Cyprus received internationally recognized FACT accreditation. CYCORD, directed by Paul Costeas, PhD, is the first and only cord blood bank in Cyprus to be recognized by FACT. The bank received accreditation on June 13, 2016, and is accredited for banking cord blood for both public and private family use.
7. Universidad Autónoma de Nuevo León, Servicio de Hematología Hospital Universitario is First Cellular Therapy Program in Mexico to Earn FACT Accreditation
Universidad Autónoma de Nuevo León, Servicio de Hematología Hospital Universitario in Mexico received internationally recognized FACT accreditation. Universidad Autónoma de Nuevo León, Servicio de Hematología Hospital Universitario, directed by David Gomez-Almaguer, MD, is the first and only cellular therapy program in Mexico to be recognized by FACT. The program received accreditation on September 6, 2016, and is accredited for Adult and Pediatric Allogeneic and Autologous Hematopoietic Progenitor Cell Transplantation, Peripheral Blood Cellular Therapy Product Collection, and Cellular Therapy Product Processing.
8. StemCyte India Therapeutics is First Cord Blood Bank in India to Earn FACT Accreditation
StemCyte India Therapeutics Cord Blood Bank, directed by Bhavin Kapadiya, MD, is the first and only cord blood bank in India to be recognized by FACT. The bank received accreditation on December 21, 2016, and is accredited for banking cord blood for both public and private family use.
9. FACT Hosts Quality Initiatives with Parent Organizations
FACT hosted a Quality Boot Camp at the annual meeting of the American Society for Blood and Marrow Transplantation (ASBMT). The goal of the boot camp program is to strengthen quality assurance activities through pre-meeting exercises and an in-person workshop. Members of the FACT Quality Committee encouraged registrants in the months leading to the in-person workshop to review specific aspects of their quality programs. Quality experts then presented important concepts and led roundtables that allowed participants to ask questions and help each other reach their goals during the boot camp. FACT also partnered with ISCT at their regional meeting to co-host sessions in the Quality and Operations Track. Presentations focused on outcome analysis from the processing perspective and the anatomy of an audit.
10. New Webinar Collaboration Between ASHI and FACT
FACT and the American Society for Histocompatibility (ASHI) began collaborating to offer webinars on transplant immunology. Establishing best practices in transplant immunology in different parts of the world requires both deep understanding of the unique challenges that face different parts of the world, and building on the experiences of well-established labs and international histocompatibility professional societies.
11. FACT Joins Standards Coordinating Body as a Charter Member
The Advancing Standards in Regenerative Medicine Act directs the Secretary of the United States Health and Human Services Agency to establish a public-private Standards Coordinating Body in Regenerative Medicine and Advanced Therapies. The Standards Coordinating Body (SCB) includes membership of the entire cross-section of regenerative medicine stakeholders. FACT was pleased to join as a charter member, and will organize its efforts as part of a consortium of non-government stakeholders that seeks to partner with one or more government organizations and regulatory agencies to coordinate consensus standards development efforts. More specifically, the SCB seeks to enable more efficient and successful clinical and commercial development of cellular/gene and regenerative medicine therapies through coordinating and prioritizing development of national/international standards for measurement assurance (among other objectives).