In the production of cell-based therapies, the quality and safety of peripheral blood mononuclear cells (PBMCs) and other raw materials from human origin (e.g., serum, growth factors, cytokines, antibodies and enzymes) must be documented and available for regulatory reviews while seeking approvals.
Although this level of documentation is not necessarily needed for basic and early-stage translational research, researchers should consider working with biospecimen providers that have these capabilities to ensure supply chain consistency throughout the drug development cycle.
The quality attributes of PBMCs and other raw materials need to be well understood, including the origin, composition and viral safety, as well as more specific requirements such as product related variance, product contact material and biological activity.(1)
The impact of supplier regulatory violations or poor quality biospecimens could be devastating for a cell & gene therapy development program, and most importantly, a patient’s second chance at life. Drug developers, therefore, should prioritize the evaluation of their PBMC suppliers’ quality and regulatory processes. Key questions to ask your supplier include:
1. What resources and expertise do you have to meet all of necessary quality and regulatory requirements?
2. Do you have a proven and reliable partner network for procuring compliant human biospecimens? How are partners assessed, contracted and audited?
3. Do you have a quality management program in place? Who on your team is responsible for this program?
4. How do you certify PBMC (or other biospecimen) characteristics and infectious disease results?
5. Do you have a board-certified pathologist on staff?
It’s essential that providers of human biospecimens have an in-depth understanding of regulatory and industry requirements to support the customer’s ongoing research efforts. PBMC products are typically procured from FDA-licensed donor centers or fall under the purview of an Institutional Review Board (IRB), International Ethical Committee (IEC), Research Ethics Committee (REC) or other similar ethical committee.
The supplier should be working with IRBs registered with either the Office of Human Research Protection (OHRP) or accredited by the Association for the Accreditation of Human Research Protection Programs (AAHRPP). In addition, providers of PBMCs should have the expertise and capacity to maintain regulatory and quality compliance in the everchanging regulatory landscape. For example, GDPR and changes to the Common Rule have significantly impacted preclinical and clinical research, and human biospecimen providers must be able to apply these changes to their relevant activities.
As a result of unprecedented research in cell & gene therapies, demand for PBMCs and other human biospecimens is growing rapidly. In response to the drive for improved quality and transparency, both government and medical professional societies have instituted regulations or accreditation programs to facilitate best practices.
As a leader in providing human PBMCs for research, Cytologics developed the Cytologics Quality System (CQS) to ensure quality and regulatory compliance in line with the best practices that have been developed over years by groups specializing in human biospecimen procurement. We take great care to validate our biospecimens meet appropriate regulations and ethical standards. If you have any questions about our collection protocols or the CQS, contact us to learn more!
(1) Papathanasiou, M.M., Stamatis, C., Lakelin, M. et al. Autologous CAR T-cell therapies supply chain: challenges and opportunities?. Cancer Gene Ther (2020). https://doi.org/10.1038/s41417-019-0157-z