As the calendar year is drawing to a close, I am writing my newsletter this month as a call to action from our FACT Community! Specifically, FACT and the member organizations of its Professional Relations Committee is seeking collaboration on comment submissions to the Food and Drug Administration (FDA) regarding its proposed rule for phasing out enforcement discretion for laboratory-developed tests. The FDA is proposing a policy under which it intends to require laboratory-developed tests to fall under the same enforcement approach as other in vitro diagnostic products, which includes regulatory approval and oversight.
FACT and FACT-accredited programs are concerned that FDA regulation of many lab-developed tests will have a tremendous and negative impact on access to care for patients (especially children and other patients with rare diseases) and will negatively impact new test development which will impact transplant and cell therapy programs. For example, lab-developed tests such as BMT chimerism tests, genetic sequencing tests, and flow cytometric tests will all be negatively impacted.
We will be providing more information in the coming weeks regarding comment submission, but the following are immediate steps that can be taken. In addition, information is provided in this newsletter which is dedicated to this issue.
- Submit a request to the FDA that the public comment period be extended to 120 days to allow adequate time to formulate responses that will fully inform the FDA of the impacts of this proposed rule.
- Review the rule and submit any initial questions you have regarding its impact to your patients to Kara Wacker at firstname.lastname@example.org.
Thank you in advance for your collaboration.
Catherine M. Bollard, MD
FACT Professional Relations Encourages Cellular Therapy Stakeholders to Request Extension of Public Comment Period for FDA Proposed Rule on Laboratory Developed Tests
The FACT Professional Relations Committee, chaired by Dr. Navneet Majhail and comprised of 13 cellular therapy stakeholder organizations, has closely followed efforts to change U.S. Food and Drug Association (FDA) regulatory oversight of laboratory-developed tests. There are many such tests that patients receiving stem cell transplantation or other cellular therapies undergo. The FDA currently practices enforcement discretion for these tests but seeks to phase out the discretion approach and begin requiring approval under medical device regulations.
After years of revisions, the Verifying Accurate, Leading-edge IVCT Development (VALID) Act, which was intended to achieve the above, failed to pass through Congress. Subsequently, the FDA announced a proposed rule available for public comment, titled, Medical Devices; Laboratory Developed Tests, which would phase out the discretionary approach via the regulatory process rather than via legislation. Comments are currently due by December 4, 2023.
It is important to emphasize that this proposed rule will have a major impact on transplantation and cellular therapy. It is necessary to carefully review the proposed rule and consider the effects it will have on patient safety and access to treatments. The Professional Relations Committee, in conjunction with member organizations’ individual efforts, will provide more information in the coming weeks. For now, there are two immediate needs for reviewing this proposed rule:
- Submit a request to the FDA to extend the public comment period from 60 days to 120 days. The current public comment period is only 60 days, with only 35 days left at the time of this writing, which many organizations do not feel is adequate time to fully study the impacts of this far-reaching proposed rule. Submitting a comment is relatively simple and can be done anonymously: go to https://www.federalregister.gov/documents/2023/10/03/2023-21662/medical-devices-laboratory-developed-tests, click on “Submit a Formal Comment,” and complete the online form.
- Send a list of laboratory-developed tests utilized by your program to the committee. A hallmark of FACT accreditation is the integration of clinical and laboratory practices. This is a great time to put that integration to action. We encourage clinicians to meet with their laboratory colleagues at their institutions to discuss what tests the laboratories perform for their patients that are considered laboratory-developed tests. Please send those tests to Kara Wacker, Strategic Planning Administrator (email@example.com) for submission to the committee or to member organization AABB, who is leading this compilation effort, at firstname.lastname@example.org.
Allowing additional time to review submitted feedback from accredited programs will help the transplantation and cellular therapy field provide the FDA with useful and relevant information to consider. Thank you for your collaboration.
by Meredith Lambe, MPA, Director of Development
For close to 30 years, FACT has globally provided fundamental education to cellular therapy program personnel pursuing FACT accreditation along with training volunteer inspectors to conduct accreditation inspections. FACT is excited to now include opportunities to connect with our industry partners to enhance education services and increase accreditation programming.
Is your organization interested in supporting FACT’s mission and vision? If so, check out our Corporate Partnership Opportunities, which include a host of ways companies can connect with FACT audiences and simultaneously support FACT services. Partnership opportunities include sponsoring FACT workshops, training programs, and webinars; recognition in the Just the FACTs newsletter, FACT e-Blasts, and FACT workshops; and supporting the FACT Quality Handbook to name just a few of the options available.
Your donation to FACT is tax-deductible to the extent allowable by law under IRS 501 (c)(3) regulations. For more information about partnering with FACT, please contact Meredith Lambe, Director of Development, at email@example.com.
Thank you in advance for your consideration to support FACT! We look forward to engaging with you in 2024!
In response to a need for a Chain of Identity (CoI) Identifier when more than one donation may be required to manufacture or deliver a given therapy, ICCBBA published the ISBT 128 Standard Chain of Identity (CoI) Identifier (ST-028) in December 2022.
Since that time, the new data structure has been implemented at many sites to document and maintain the chain of custody for patient-specific cell therapy manufacturing. An implementation guide has now been drafted to provide guidance on how the CoI Identifier can be utilized in various scenarios. ICCBBA is soliciting public comment to further strengthen the usefulness of the draft implementation guide.
We encourage your participation in the public comment period. The authors have requested comments to be submitted by November 20, 2024.
Comments should be directed to IG050@isbt128.org.
by Stacy Freeburg, Accreditation Coordinator
They say the only thing constant is change. This is true for healthcare facilities, which are often rearranged, remodeled, or built new to meet the shifting needs of patients. What does not change, though, is an accredited organization’s responsibility to maintain compliance with FACT Standards throughout a move of cellular therapy-related activities.
FACT used to automatically require an interim inspection of new or relocated facilities so that compliance with Standards could be verified. In 2013, after reviewing several years of relocation inspection results, the FACT Accreditation Committee determined that such inspections are not always required because sufficient information could be provided.
The current policy requires accredited organizations to update FACT within 90 days of a facility relocation and submit specific documentation to demonstrate compliance with FACT Standards. This includes temporary moves. The Chair of the FACT Accreditation Committee reviews the documentation and determines if a relocation inspection is necessary. It is recommended that organizations notify their assigned Accreditation Coordinator in advance if a relocation is planned, and organizations should expect to provide the following information, as applicable:
- A description of the new facility.
- An explanation or reason for the move.
- If the move is temporary, include a timeline for when services will resume at the current location and the revalidation plans for the processes, facilities, and equipment.
- Mailing and physical address of new and old locations, including building, floor, wing, room number.
- Include the distance between the old location and the new location, if applicable.
- Indicate if the new location has been inspected by FACT previously.
- Date operations started at new facility and type of processes.
- The date when the move occurred.
- A risk assessment of the move, including how adequate space and protection from airborne microbial contamination and provision of adequate medications, blood products, and other critical services, were maintained during the relocation.
- A detailed floor plan (with clearly marked rooms, exits, restroom facilities, any isolation rooms, storage areas, irradiators, etc.) and the identification of the new location.
- A summary of any changes to the Quality Management Plan, if applicable.
- A summary of the SOPs that were created or revised because of the relocation, as applicable, and provide an example of a revised SOP.
- A summary of staffing changes, if applicable.
- Include a summary of the validations, revalidations, verifications, or qualifications that were performed due to the relocation.
- Submit a completed example for one of the following: validation, revalidation, verification, or qualification.
- Include a schedule for completing these assessments if not completed at the time of submission.
It is with great sadness that we remember Susan Pinkard, who passed away in August 2023. Sue was a long-time and dedicated FACT inspector. She served as an inspector for 10 years and performed 18 inspections. Sue particularly demonstrated her commitment to FACT following the COVID-19 pandemic, after which she performed several inspections once they were resumed.
As described in Sue’s obituary she loved her family and devoted her career to helping bone marrow transplant patients. To read Sue’s full obituary, visit https://www.springgroveobituaries.org/obituaries/Susan-Lee-Pinkard?obId=28888026.
A new nonprofit, TrueTrials, announced its online platform for listing U.S.-based and FDA-approved clinical trials. Considering itself the cell-based science version of a Good Housekeeping seal of approval, the platform is intended to provide patients and families with an easy resource to find approved trials for various indications.
TrueTrials will list only clinical trials that are authorized by the FDA, and its team verifies every 60 days that listed trials are still enrolling patients. Therefore, the database remains current and relevant for patients. The website also includes educational content and currently includes videos from an interview with Peter Marks of the FDA.
For more information regarding how patients can use TrueTrials, or how to list enrolling trials on the platform, see the TrueTrials website.
by Jamie Price, MS, BA, Director of Education
Save the Cord Foundation is hosting World Cord Blood Day on November 15, 2023. FACT is honored to be an inspiring partner again this year to help raise awareness about the current and future uses of cord blood stem cells.
World Cord Blood Day was created to expand cord blood education worldwide. Cord blood transplants exceed 40,000 globally, and stem cells from cord blood are currently used to treat more than 80 different types of cancer, including leukemia, lymphoma, and sickle cell anemia. Cord blood and cord blood stem cells are also utilized in clinical trials for many indications including a variety of diseases and conditions.
As an inspiring partner, and in anticipation of World Cord Blood Day on November 15th, FACT invites the public to participate in the Save the Cord Foundation’s free virtual conference. Register online today at https://www.worldcordbloodday.org/ in honor of World Cord Blood Day, November 15, 2023. #WorldCordBloodDay | #WCBD23