Many of you in North America are tired of winter and cold, and we have some hot topics in this newsletter to get your circulation going. One topic is FACT’s contribution of comments to the CMS national coverage determination for CAR T-cell therapies. We hope the coverage will be clarified in a manner that covers the costs to the institution beyond the purchase of the product. Also, I invite all of you to consider volunteering for the Standards Committee for the upcoming revision cycle. This is very much a working committee, but there is no better way to know what’s coming and contribute to the community than through the making of clinically important standards!
As always, there are many educational events throughout the year. Review FACT’s educational calendar and register for an event today.
I look forward to seeing many of you at the ISCT Annual Meeting in Melbourne!
Articles in this volume include:
- Feedback Requested: FACT-JACIE Standards
- Trending Clinical Outcomes
- College of American Pathologists Recognized as an Accrediting Organization Providing Histocompatibility Services Appropriate for Hematopoietic Cellular Therapy Transplant Patients
- FACT Submits Comments to CMS Regarding Proposed National Coverage Determination for CAR T-Cell Therapies for Cancer
- FACT Remote Director Policy
- Additional FACT Volunteer Opportunities
- Education Events
Dennis Gastineau, MD
By: Andra Moehring, MHA, FACT Standards Development Manager
Preparation of the Eighth Edition FACT-JACIE International Standards for Hematopoietic Cellular Therapy Product Collection, Processing, and Administration will commence this summer. Final publication is expected in March 2021.
FACT and JACIE invite you to complete a short survey focusing on general concepts of interest. You are also encouraged to promote this survey in your national or regional professional society. Responses will be accepted through June 1, 2019.
The results of this survey will be reported to the Standards Steering Committee for review and consideration.
By: Kara Wacker, MBA, FACT Strategic Planning Administrator
Clinical outcome analysis might have started gaining renewed attention in recent years, but FACT has required such analysis since its first Standards in 1996. This puts the HPC transplant field at an advantage as more stakeholders and patients are evaluating health care institutions in the context of actual clinical outcomes. Transplant programs that have voluntarily complied with FACT Standards over the years have outcome data that can be useful not just for retroactive investigations but for anticipating lower-than-expected outcomes in the future . . . and addressing them before undesirable outcome trends result.
When programs are required to submit a corrective action plan to address lower-than-expected one-year survival (per the FACT-JACIE Hematopoietic Cellular Therapy Standards), a common question asked of FACT is, “what time frame should my plan address?” The time frame should include current data, but should also go back in time far enough to detect any trends.
For example, autologous-only or non-U.S. programs often use the C.W. Bill Young Cell Transplantation Program’s U.S. Patient Survival Report or the Be the Match/NMDP Disease-Specific HCT Indications and Outcomes Data as comparative data sources. These reports are by year, but a single year is not long enough to adequately evaluate outcomes to detect problems.
Some allogeneic programs in the U.S. assume the time frame is limited to that used in the Transplant Center-Specific Survival Report published annually by the Center for International Blood and Marrow Transplant Research (CIBMTR). These time frames consist of three calendar years, with the last year being two years prior to report publication. The FACT Clinical Outcomes Improvement Committee does expect that these years be included; however, programs are not limited to this time frame. In fact, survival from before the time frame in the CIBMTR report will help programs detect when a declining trend began. Data from years following the CIBMTR report illustrate a complete picture of the current survival rates of the program, and can be used to determine effectiveness of corrective actions. Such data should readily be available from internal outcome analyses required by the Standards. They can also be obtained from CIBMTR’s Data Back to Centers (eDBtC) resource.
A FACT-accredited program shared a trending tool with FACT, and gave us permission to anonymously make it available to other programs on the FACT website. This Microsoft Excel-format Risk and Non-Risk Adjusted Survival Data and Box Graph has fields for entering figures from multiple CIBMTR reports over time (such as number of first-transplant patients, risk-adjusted survival, and endpoints of the expected range), plus non-risk adjusted one-year survival rates from internal data or eDBtC. After inputting this data, a graph on the second sheet is automatically populated.
An example is below. In this example, the program met clinical outcomes in both the 2013 and 2014 cohorts (2009-2011 and 2010-2012, respectively). However, the program’s survival rate declined. By the next report in 2015, the program did not meet expected one-year survival. As a program’s risk-adjusted actual survival declines and gets closer to the lower limit of the 95% confidence interval, it can begin evaluating causes of death of its patients and implementing corrective actions before one-year survival becomes lower than expected.
If survival rates are declining, questions a program may want to ask include:
- Is there a difference in causes of death, or have any specific causes increased?
- Did disease/patient case mix change?
- Are protocols still being followed?
- Are there any changes in psychosocial patterns?
- Do records adequately follow patients?
- Do protocols need updated to current science?
Programs often fall in and out of expected survival ranges, and they should monitor how close their survival rate is to the lower limit. Avoid complacency just because survival was within the expected range; make a genuine effort to robustly complete required internal outcome analyses to detect problems before the CIBMTR report does. After all, we all know the real goal isn’t a score of “0” on the report. It is improving patient survival.
College of American Pathologists Recognized as an Accrediting Organization Providing Histocompatibility Services Appropriate for Hematopoietic Cellular Therapy Transplant Patients
By: Andra Moehring, MHA, FACT Standards Development Manager
The Seventh Edition FACT-JACIE International Standards for Hematopoietic Cellular Therapy Product Collection, Processing, and Administration require (B2.14) “Clinical Programs performing allogeneic transplantation shall use HLA laboratories that are … accredited by the American Society for Histocompatibility and Immunogenetics (ASHI), the European Federation for Immunogenetics (EFI), or other accrediting organizations providing histocompatibility services appropriate for hematopoietic cellular therapy transplant patients.” Similar standards exist in the FACT Common Standards (B2.9), the NetCord-FACT Cord Blood Standards (B5.6), and in the FACT Immune Effector Cell Standards (B2.9).
To facilitate a fair and objective assessment of appropriateness, the HLA Typing Accreditation Committee of FACT, JACIE, NetCord, and NMDP developed Guidelines for Histocompatibility Typing Standards and Accreditation Programs. These Guidelines will be reviewed every three years and revised appropriately. Each applicant accrediting organization is required to submit documentation to FACT for review by the HLA Typing Accreditation Committee that demonstrates appropriateness of its standards and accreditation program for the relevant population.
The College of American Pathologists (CAP) submitted its documentation to FACT for review. During the review and feedback process, it was clear that CAP had a significant commitment to being recognized by FACT as an accrediting organization providing histocompatibility services appropriate for hematopoietic cellular therapy transplant patients.
The FACT Board of Directors voted to approve CAP as an accrediting organization providing histocompatibility services appropriate for hematopoietic cellular therapy transplant patients. With this approval, CAP will be accepted in FACT Accreditation applications and included in FACT Standards. CAP will include the HLA Typing Accreditation Committee during its review and revisions of Standards and Checklists on an annual basis to ensure continued appropriateness for inclusion in FACT’s Standards and Accreditation Program.
FACT Submits Comments to CMS Regarding Proposed National Coverage Determination for CAR T-Cell Therapies for Cancer
By: Kara Wacker, MBA, FACT Strategic Planning Administrator
The United States Centers for Medicare & Medicaid Services (CMS) released a proposed decision memo that outlines coverage for CAR T-cell therapies for cancer using a National Coverage Determination (NCD) with Coverage with Evidence Development (CED). FACT responded to this proposal on March 15, 2019. Comments were a result of input from a variety of stakeholders, including the FACT Board of Directors and Immune Effector Cell Task Force, FACT-accredited programs, FACT inspectors, professional societies, data registries, manufacturers, and payers. We commend the cellular therapy field for its collaborative efforts to provide feedback that would be helpful to CMS as it seeks to provide beneficiaries access to CAR T-cells, and we appreciated this opportunity to provide feedback.
Highlights of FACT’s comments include:
- Suggestion that coverage is provided based on Food and Drug Administration (FDA)-approved product labels to allow coverage as therapies advance.
- Support for the use of the Center for International Blood and Marrow Transplant Research (CIBMTR) as the data registry, and suggestion to certify the registry prior to finalization of the proposed decision memo.
- Request for clarification on the utility of quality of life data for the stated purposes.
- Clarification regarding the scope of various sets of FACT Standards.
- Agreement that hospitals should meet FACT requirements to promote safety and efficacy of CAR T-cell therapies; increase harmonization across requirements of regulators, payers, and manufacturers; and reduce burden on hospitals.
By: Cassie Kennedy, BS, FACT Accreditation Coordinator
All editions of FACT-JACIE International Standards for Hematopoietic Cellular Therapy, NetCord-FACT Standards for Cord Blood Banking, FACT Common Standards, and FACT Immune Effector Cell Therapies require that there be Directors and Medical Directors of the program, bank, and participating facilities. Standards define specific qualifications and responsibilities, but are silent on residency requirements and on the distances among accredited programs or facilities for which one individual serves as director.
Remote Directors and Medical Directors are those directors with professional responsibilities in more than one metropolitan geographic area, or those whose residence is outside of the metropolitan geographic area of the accredited facility. The FACT Accreditation Committees have increasingly noted instances of remote directors who purportedly are responsible for two or more accredited facilities outside of a single metropolitan area, hundreds of miles apart, or in another country. Most commonly, these facilities are apheresis centers, processing facilities, or cord blood banks; however, clinical program directors also have raised similar questions. The concern is whether or not the Collection and Processing Facility Directors who oversee multiple sites in various states or regions, or who live in states or countries distant from the accredited facilities they oversee, are present enough to fulfill the role as intended in the Standards.
The Remote Directors Task Force
The Task Force was constituted in July 2017 to develop recommendations regarding minimal on-site requirements, or suitable alternative arrangements, for Directors and Medical Directors of FACT-Accredited Apheresis Collection Facilities, Processing Facilities, and Cord Blood Banks.
The Task Force membership was diverse, including directors, medical directors, and non-directors in Cellular Therapy Programs (Clinical, Collection, and Processing) and Cord Blood Banks.
The Task Force reviewed the Director and Medical Director responsibilities as detailed in the Standards. They considered examples of facilities wherein citations were generated at the on-site inspection due to questionable level of director involvement or known location of the director in another state. They also considered the various infrastructures that exist in accredited facilities; the presence or absence of supporting, ancillary, or professional personnel in the facility, including cross-coverage of various functions among the clinical, collection, and processing staff members; and the impact of technology. The Task Force was not able to come to a consensus minimum number of hours or days that any director or medical director must physically be present in a facility to meet the standard.
Guidance for Remote Directors and Medical Directors
Responsibilities for remote directors do not differ from the responsibilities of any director, however, there may be more challenges in completion and documentation of these responsibilities. The following are requirements for remote directors:
- A director must be fluent in the language of the facility and must meet the minimum credentials, training, experience, competency, and continuing education requirements as defined in the current edition of Standards.
- A director is responsible to lead the accredited program, facility, or bank and to provide oversight of the services, personnel, products, and procedures.
- A director is expected to be actively engaged in the decision-making process, policy and procedure development, and quality management activities. This involvement must be documented.
- When a director is physically not present at the facility site, there must be a qualified designee named and documented to manage those responsibilities that require immediate or in-person attention. Further, all critical director functions must be covered.
- A qualified designee must meet minimum director qualifications for the delegated function, and have a defined scope of authority and activity.
- Specific responsibilities of each director and medical director type are defined in the applicable set of Standards and are summarized in the table below. Documentation of director involvement in these responsibilities must be available on-site for review.
Documentary evidence of the remote director’s specific involvement in leadership and oversight of the program, in addition to performance of designated responsibilities, must be available on-site for review by the inspector. Examples of documentation include, but are not limited to:
- Meeting minutes
- Record review
- Personnel review
- SOP review and approval
- Donor or recipient management
- Investigation report review
- Qualification/validation studies: plan and final report review and approval
- Planned deviation pre-approval
- Product release authorization
|Cellular Therapy Director Type||Applicable Standards (7th Edition)|
|Clinical Program Director||B3.1.3, B3.1.4, B3.1.5, B18.104.22.168, B4.1.1, B4.2.1, B22.214.171.124, B126.96.36.199, B4.13.3, B188.8.131.52, B4.17, B4.18, B5.3.8, B5.3.9, B5.7|
|Marrow Collection Facility Medical Director||CM184.108.40.206-CM220.127.116.11, CM5.3.8, CM5.3.9, CM5.7, CM7.2.2, CM7.2.3|
|Apheresis Collection Facility Director||C3.1.2, C4.1.1, C4.2.1, C18.104.22.168, C4.13.3, C22.214.171.124, C4.17, C4.18, C5.3.8, C5.3.9, C5.7, C7.2.2, C7.2.3|
|Apheresis Collection Facility Medical Director||C3.2.2, C126.96.36.199, C5.3.8, C5.3.9, C5.7|
|Processing Facility Director||D3.1.2, D4.1.1, D4.2.1, D188.8.131.52, D4.13.3, D184.108.40.206, D4.17, D4.18, D5.3.7, D5.3.8, D5.7, D7.2.2, D7.2.3, D8.1.1, D220.127.116.11, D8.7.1, D8.9, D11.1.1, D18.104.22.168, D22.214.171.124, D11.3.6, D126.96.36.199|
|Processing Facility Medical Director||D3.2.2, D4.9.4, D188.8.131.52, D5.3.7, D5.3.8, D5.7, D8.1.7, D8.10, D184.108.40.206, D220.127.116.11, D12.1.4|
|Cord Blood Director Type||Applicable Standards (6th Edition)|
|Cord Blood Bank Director||Appendix I, B2.11.5, B18.104.22.168, B22.214.171.124, B126.96.36.199, B188.8.131.52, B184.108.40.206, B2.13.1, B2.14.1, B220.127.116.11, B2.19, B3.2.3, B18.104.22.168, B6.2.5, B12.2, D6.4.2|
|Cord Blood Bank Medical Director||Appendix I, B2.11.5, B22.214.171.124, B126.96.36.199, B188.8.131.52, B184.108.40.206, B5.5.5, E4.2, E4.3.1|
|Cord Blood Collection Director||Cord Blood Collection Director|
|Cord Blood Processing Facility Director||Appendix I, B2.11.5, B220.127.116.11, D9.2.7|
By: Andra Moehring, MHA, FACT Standards Development Manager
The Foundation for the Accreditation of Cellular Therapy (FACT) has several committees to help FACT achieve its vision of being the premier organization setting standards and awarding accreditation to programs in the evolving field of cellular therapy.
In addition to other standing committees, FACT has a special committee for which we are seeking volunteers. We invite you to review the charge for the FACT Standards Committee and either nominate yourself or a colleague to serve as a committee member.
The FACT Standards Committee drafts standards and guidance for cellular therapy product collection, processing, and administration; and cord blood collection, banking, and release for administration, using evidence-based science and considering evolving uses of cellular therapy.
The committee’s goal is to provide clear and user-friendly FACT standards and effective transition between editions of Standards.
- 3 year term
- Minimum of 2 three-month periods with weekly or bi-weekly teleconferences
- Complete initial edits prior to the public comment period
- Review of public comments submitted
- Ongoing participation until the development of the next edition of Standards
The majority of the hard work is completed during subcommittee discussions that take place several months before the actual publication of the Standards. The experience and expertise of our volunteer subcommittee members is invaluable to the development of each edition of Standards.
Standard Development Schedule
FACT currently has four sets of Standards, with multiple subcommittees (e.g., Clinical, Collection, Processing, Quality Management, Immune Effector Cell) under each set. The Standards Development process for each upcoming edition of Standards is below:
- FACT-JACIE International Standards for Hematopoietic Cellular Therapy Product Collection, Processing, and Administration (July 2019)
- FACT Standards for Immune Effector Cells (July 2019)
- FACT Common Standards for Cellular Therapies (May 2020)
- NetCord-FACT International Standards for Cord Blood Collection, Banking, and Release for Administration. (January 2021)
Volunteers are an essential resource for FACT. Our success and accomplishments are directly attributable to the selfless commitment of our knowledgeable volunteers. FACT’s volunteers bring new ideas and energy to the Foundation. Your volunteering makes a difference in the organization and the cell therapy community.
Please submit your interest in serving as a committee member along with a description of your qualifications to Andra Moehring, FACT Standards Development Manager, at firstname.lastname@example.org by May 1, 2019.
FACT Cellular Therapy Collection Workshop at 2019 Annual ASFA Meeting
May 15, 2019
Join FACT on Wednesday, May 15, at the Hilton Portland Downtown in Portland, OR, for a workshop designed to explain the requirements for FACT accreditation of cellular therapy programs, with special emphasis on apheresis collection facilities.
FACT representatives will be in attendance to discuss cellular therapy collection challenges, provide examples on how to maintain quality when faced with different collection protocols, and present a common citations overview. This workshop also includes inspector trainee sessions on how to perform a FACT inspection from start to finish.
FACT Cellular Therapy & Cord Blood Inspection at Accreditation Workshop at the 2019 Annual ISCT Meeting
May 28, 2019
This workshop incorporates instruction on the process of accreditation, FACT Standards requirements, and practical applications such as risk assessment and transportation issues related to cellular therapy and cord blood banking. Several hot topics include quality management in novel cell therapies, written agreements, and occurrence management. These sessions are designed to help applicants navigate the evolving changes in the field. The agenda also includes training for new inspectors with real-world examples and exercises to guide them through the inspection process. This is a must-see event for the following audiences:
• Blood and marrow transplant (BMT) programs
• Cord blood banks
• Cell processing laboratories
• Physicians, scientists, and principal investigators
• Unit supervisors, laboratory managers, administrators, nurses, and medical technologists
• Quality managers
The day includes a general track for attendees with separate breakout sessions for cellular therapy and cord blood participants. Inspector trainees are required to attend the inspector orientation track.
ISCT-FACT Quality Boot Camp at 2019 ISCT Annual Meeting Pre-Conference Day
May 29, 2019
Is your cell processing facility FACT accredited or looking to comply with FACT standards? Strengthen your quality assurance program through this interactive workshop with FACT experts and real world applications.
Focused on issues identified by FACT-compliant transplant programs, these sessions will include activities and discussion in Document Control, Incorporating IECs into your Quality Plan, Performing Risk Assessment: Real Life Applications, Process Validation, and Evaluating and Reporting Audit Results. Come prepared with your quality program questions and leverage quality experts to identify process improvement opportunities you can apply at your organization.
FACT Cellular Therapy Inspection and Accreditation Workshop at CBMTG Annual Meeting
June 5, 2019
Calgary, Alberta, Canada
The blood and marrow transplantation field has been a leader in voluntarily improving quality through FACT accreditation. This workshop will address important topics in the cellular therapy field including:
• Overview of FACT Accreditation Processes
• Common Citations Overview
• Building Quality into Clinical Immune Effector Cell Therapy Programs
• Using the Tracer Methodology to Assess Compliance with FACT Standards
• Improving Data Management Through Follow-Up
• FACT Process for Reviewing Clinical Outcomes
New Webinar Announced in Quality Management Series 9: Auditing
The Quality Management Series Module 9 includes four sessions focused on auditing. The first session, Auditing: Planning and Executing, was held on January 23, 2019. Leigh Poston, BMT Program Quality Management Coordinator at UF Health Shands Hospital, discussed designing audit strategies, identifying specific audits to perform, creating an audit schedule, determining the scope of the audit, and executing the audit. View on-demand webinar
The second session, Auditing: Summarizing and Evaluating the Data, was live on March 6, 2019. Ed Brindle MSc, MLT, Quality Manager at UHN Blood and Marrow Transplant Program, discussed writing the audit report, determining WHY the errors occurred, and assigning appropriate corrective action and preventive action. View on-demand webinar
The third webinar in the QM series 9: Audits, titled Performing follow-up to assess the effectiveness of the actions implemented on correcting the errors, will be on June 11, 2019 at 11:00 am ET. Gordana Svajger, Quality Compliance Program Manager at The Hospital for Sick Children Blood & Marrow Transplant / Cellular Therapy Program, will identify methods for verification of corrective actions, interpret when corrective actions are effective, and determine when to close an audit. View webinar details and register
The last session in the series will be, A Program’s Perspective: Auditing. The date and time will be announced when determined.
Register for the entire module to receive a 15% discount! QM Series Module 9: Auditing
WMDA-NetCord & FACT Cord Blood Day at 2019 Cord Blood Connect International Congress
September 12, 2019
Join your cord blood colleagues for a day of presentations from renowned cord blood experts at the 2019 WMDA-NetCord & FACT Cord Blood Day! The event will be held in Miami, Florida on September 12, 2019, in conjunction with the Cord Blood Connect International Congress.
The WMDA-NetCord & FACT Cord Blood Day will highlight recent advances in cord blood transplantation and include global perspectives of cord blood transplants, common FACT-NetCord inspection citations, and more. The afternoon consists of two breakout sessions: a FACT inspector training track and a WMDA-NetCord & FACT cord blood quality boot camp track. We encourage banks to send multiple personnel to attend the various tracks as well as enlist a member to become a FACT inspector.
This is an excellent opportunity to network with the cord blood community and learn from experts in cord blood banking. Registration fees are waived for FACT active and trainee inspectors. Apply to be a FACT inspector!
FACT Cellular Therapy Inspection and Accreditation Workshop at the ISCT North America 2019 Regional Meeting
September 12, 2019
This training workshop is designed to explain the requirements for FACT accreditation of cellular therapy programs. FACT representatives will be in attendance to clarify the intent of the FACT-JACIE Standards, provide inspectors tips for conducting inspections, and assist programs in organizing and preparing for the accreditation process.
Two tracks are available for this workshop. The Inspector Track will provide FACT inspectors guidance on how to assess compliance with the Standards. The Applicant Track will give programs and facilities insight into how to comply with the Standards and demonstrate compliance.