FACT participates in several working groups of the Standards Coordinating Body (SCB), including the Cell Collection Standards for Cell and Gene Therapies group. The SCB website introduces this group as follows: “Because other advanced fields with similar biological products (e.g., bone marrow and apheresis) have established collection best practices, the regenerative medicine community is eager to leverage those practices to create an international collection process standard that will help ensure the consistency of the critical quality attributes of cells collected for regenerative medicine products.”
With interest and participation from industry and the United States Food and Drug Administration (FDA), the efforts of this group may impact FACT-accredited Apheresis Collection Facilities and we strongly encourage participation of apheresis professionals given their direct experience and expertise in cell collection.
Through the work of this group, SCB has successfully obtained agreement from the Parenteral Drug Association (PDA) to draft standards on cell collection. Although industry is the intended audience for these standards, requirements may be promulgated through the requirements that clinical trial sponsors and commercial manufacturers expect of apheresis collection sites in their networks.
The PDA is recruiting volunteers to draft the requirements, currently referred to as “Apheresis Collection for Cell and Gene Therapy Products,” until July 16, 2021. “The goal is to create a standard document outlining recommendations for product manufacturer/sponsor requirements for apheresis cell collection. It will also create a standardized template for the leukapheresis manuals/SOPs written by product manufacturers/sponsors for the apheresis centers. This combined effort will work to reduce the burden upon the collection centers and in doing so, reduce errors.”
FACT encourages participation from accredited Apheresis Collection Facilities. According to the PDA, volunteers must have some subject matter expertise, and be willing to help write/contribute to this standard. Applicants should apply by contacting the PDA Standards Manager at email@example.com.
Apply to the PDA technical team for this standard by July 16, 2021!
To Our Colleagues in Industry:
The SCB is asking industry groups and manufacturers to share their apheresis collection requirements and guidance to help with the creation of the new Parenteral Drug Association (PDA) standard effort, PDA 08-202x: Apheresis Collection for Cell and Gene Therapy Products.
For this effort to be successful, the PDA working group will need a clear view of what industry groups and manufacturers currently require, so that it can fit the standard as close to common industry practices as possible. To gain that insight, please fill out this short survey. The data collected will only be viewed/used in aggregate and will not be used outside of the standards effort. Please fill out this survey at your earliest convenience.
FACT accreditation is a credential that has its greatest value in the peer-to-peer exchange of ideas to confirm and improve quality in patient care and laboratory services. Successful accreditation is awarded with two tangible items: an Accreditation Certificate and a final Accreditation Report. Although the certificate gets more prominence, often framed and mounted as a visual symbol of quality, the final report is a detailed accounting of what accredited programs implemented to reach compliance with all FACT Standards.
Each Accreditation Report provides important information related to an accredited program’s journey to initial or renewal accreditation. The final report includes:
- The name and location of the program
- The date of the on-site inspection
- The clinical, collection, processing, and/or banking sites and services associated with the accreditation
- Key personnel
- Identification of the FACT inspection team
- A description of the program
- Deficiencies and variances cited by the inspection team and approved by the Accreditation Committee
- Required responses to provide evidence of correction of each deficiency or acknowledgement of each variance
- The program’s responses documenting completion of corrective actions and compliance with Standards
- FACT’s response, or determination regarding the adequacy of the program’s corrective action
- Details of any additional follow up reports or documentation due to FACT
After completing the rigorous accreditation process, programs may debrief the contents of the Accreditation Report and then file it away, turning attention to its many other important duties until annual reporting is required. However, the final report is a valuable tool that can be used throughout the accreditation cycle.
Programs that have corrected deficiencies and submitted adequate evidence of correction and compliance with standards should be very proud of the work they have invested into improvement. Lessons learned throughout this process can be applied to the entire program. For example:
- How will we evaluate our corrective actions to confirm long-term effectiveness?
- What other strengths and weaknesses did we discover while correcting deficiencies?
- Are there further root cause analyses we should perform to prevent similar deficiencies?
- Can we apply the same process to evaluate other aspects of our program?
Ideas generated from this review of the Accreditation Report may be useful to maintaining the Quality Management program via audits, process development, personnel training, and other quality concepts.
External Confirmation of Quality
Apheresis collection facilities, cell processing facilities, and clinical services are each important in the context of blood and marrow transplantation, where accreditation covers the entire scope of the therapy from patient selection and treatment, through product selection, collection, processing, and administration. These same services participate in clinical trials often including products developed and manufactured by industry partners. These facilities recognize the importance of standards, have achieved voluntary FACT accreditation, and have played a major role in delivering new products to patients. They have used their quality programs and processes to quickly adapt to increasing numbers of products, procedures, and manufacturers. However, facilities have individually and collectively expressed concern regarding the growing number of on-site audits. The increased burden of preparing for, participating in, and responding to audits requires more resources to be diverted to these activities rather than toward patient care.
Commercial manufacturers who produce cellular therapy products for clinical trials or under an approved regulatory license may wish to leverage FACT accreditation to streamline audit activities, verify quality practices, and reduce the burden on health care entities. To do this, manufacturers may request a copy of the final FACT Accreditation Report. FACT does not provide Accreditation Reports to any entity except the accredited program. Accredited programs are not restricted from sharing these reports and may choose to provide them to manufacturers leveraging FACT accreditation, however, they are not required to do so.
Accredited programs or their hospital administration may be hesitant to disclose deficiencies cited during an on-site inspection. However, FACT accreditation is a voluntary, peer-based quality improvement process, during which some deficiencies can be expected at any program. The documented deficiencies demonstrate the rigor of the inspection process. The final report includes the description and documentation of the program’s corrective action, FACT’s determination of the adequacy of the response, and any additional follow up that may be indicated. Responses to deficiencies are an opportunity to proudly share a program’s process improvements. Programs should inquire of any commercial manufacturer regarding the confidentiality of information they provide to increase confidence in the use of the FACT report.
If a program chooses to provide the final Accreditation Report to a commercial manufacturer, it will likely be able to participate in an abbreviated site audit because the Accreditation Report can be used to confirm the quality systems in place meet FACT Standards. If a program chooses not to provide the report, it may be required to undergo a full site audit that will include requirements duplicative to the FACT Standards.
ISBT 128 Labeling Standard for Cells Collected for Cellular Therapy Manufacturing: Comments Due October 18th
Thanks to the teamwork of several stakeholders in apheresis cell collection, the field is one step closer toward label standardization. Comments are needed in the most recent draft ISBT 128 label published by ICCBBA by October 18, 2020.
As reported in previous Just the FACTs newsletters, a multi-stakeholder working group led by the Standards Coordinating Body and the Deloitte NextGen Industry Working Group, consisting of industry, registries, accrediting organizations, technology providers, and apheresis and quality subject matter experts, released a draft standard labeling proposal for collected cells.
Public comments following the release of that draft indicated support for ISBT 128 labeling for starting material intended for further manufacturing. After reviewing these comments, ICCBBA has released a draft Standard for Labeling of Collection Products for Cellular Therapy Manufacturing.
This Standard builds on existing ISBT 128 standards, including compatible label dimensions and essential traceability information, but also accommodates sponsor/manufacturer information. As stated by the ICCBBA website, “it is for use only in situations where the sponsor/manufacturer has adopted this standard and has provided the necessary information to populate the sponsor/manufacturer section of the label.” Although the standard was written with apheresis in mind, the label could be used on products collected from other sources and intended for further manufacturing.
The Cellular Therapy Community Corner provides a forum for the cell therapy community that includes educational opportunities, recent publications, and upcoming events.
ASGCT Debuts Patient Education Program
The American Society of Gene & Cell Therapy (ASGCT) announced the first release of the Society’s Patient Education program, a new initiative for 2019. Designed by ASGCT committee volunteers in coordination with patient advocacy groups, the new patient-centered portal is designed to educate and inform patients, families, and the public on the status and promise of gene and cell therapies.
The following Sessions are now live on ASGCT.org:
Gene Therapy 101, which includes the following information:
Disease Treatments, which includes the following information:
- Spinal Muscular Atrophy
- X-Linked Myotubular Myopathy
- Leukodystrophy (coming February 15)
- Blood Disorders (coming February 22)
- Inherited Retinal Disorders (coming February 28)
All topics ultimately provide patients and patient advocates with video, text, and infographic resources to share with various stakeholders. ASGCT encourages people to share these resources with their friends and family.
FDA Outlines Planned Policies to Advance Development of Safe and Effective Cell and Gene Therapies
Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research
On January 15, 2019, FDA announced its plans to provide policy guidance and advance its drug development network over the coming year in response to increases in cell and gene therapy investigational new drug (IND) applications. The FDA anticipates receiving more than 200 INDs per year by 2020 and approving 10 to 20 cell and gene products a year by 2025.
The full statement outlines FDA’s policy agenda as it relates to these technologies.
Find a clinical trial through the Jason Carter Clinical Trials Program Offered by The National Marrow Donor Program®/Be The Match®
The National Marrow Donor Program®/Be The Match® is offering a new program, The Jason Carter Clinical Trials Program, designed to help patients with blood cancers or blood disorders and their families find and join clinical trials. Funding was provided by the Carter family, in memory of their son and brother, Jason Carter. This free program offers:
- One-on-one telephone support and information from a clinical trial nurse to help patients and families navigate their clinical trial search.
- Easy-to-use, web-based search tool to find relevant clinical trials: JasonCarterClinicalTrialsProgram.org
- Easy-to-understand educational resources for patients and families to learn about cancer treatment options and clinical trials.
On the JCCTP.org website, there are patient-friendly clinical trial descriptions related to leukemia, lymphoma, MDS, aplastic anemia, and more. Treatments in some of these clinical trials include blood or marrow transplant, but many others trials include the latest cell therapies such as CAR T and immunotherapies.
Patients can also apply for financial assistance from the Drs. Jeffrey and Isabel Chell Clinical Trials Travel Grant. This grant works to offset the cost of travel related to clinical trial participation. Grant eligibility information and application are available on JasonCarterClinicalTrialsProgram.org.