CBA Testifies on Regulation of Cellular Products, Therapies

Cord Blood Association Issues Guidelines for Zika Virus Screening

Issued:  September 13, 2016

The unique characteristics of cord blood and related-tissue products and therapies should be acknowledged in the regulation of cellular products and regenerative medicine, according to testimony presented by the Cord Blood Association at an FDA public hearing.

President Joanne Kurtzberg, MD, told how regulations created for pharmaceutical manufacturing, when applied to cord blood, can inhibit innovation and add to procedural burdens and costs, yet often provide few safety and efficacy benefits.

Among key points in the testimony: 

  • The current regulations, designed mostly for the manufacture of drugs, are insufficient for cellular therapies.
  • Cord blood is not a bag of stem cells.  Although it does contain small numbers of blood stem cells, the majority of cells are differentiated blood cells.  Some of these other cells have therapeutic value, but do not act through engraftment, tissue integration or differentiation.  Rather they are effector cells, acting through parancrine signaling.  As such, their action should be considered as homologous.
  • The designation of a tissue as minimally or more-than-minimally manipulated should be risk-based, with consideration of clinical indication, route of administration and complexity of product manufacturing.  If cells are prepared aseptically and only exposed to FDA approved-for-human-use reagents and devices, manufacturing should be considered minimally manipulated.
  • Current rules governing autologous cells or tissue obtained from first-degree and second-degree relatives are outdated.  Related HLA-identical and haplo-identical products should be among the exempt products under Chapter 21, Section 1271 in the Code of Federal Regulations.
  • The FDA should consider a pathway for cellular therapies similar to that already established for hematopoietic stem cell and solid organ transplantation.  One way to bring safe and effective therapies to patients more quickly would be to encourage development of therapies in accredited facilities, under investigational new drug (IND) monitoring when indicated, and reported to a registry such as the Center for International Blood and Marrow Transplant Research (CIBMTR). 
  • Excessive environmental monitoring adds little, if any, value to the manufacture of cord blood products that is performed in a closed system in which appropriate qualification testing is performed and specifications are met.
  • Requiring stability protocols for stored cord blood does little if anything to enhance safety and efficacy, but does sacrifice unique cell products that cannot be replaced.  

The purpose of the FDA public hearing was to receive comments on four draft guidances on the regulation of human cells, tissues and cellular and tissue-based products.  

Cord blood was the first hematopoietic stem cell product to be regulated by the FDA.  To date, seven public cord blood banks have successfully obtained biologics license application (BLA).  “Lessons learned from the cord blood BLA should inform regulation of other cell therapies going forward,” Dr. Kurtzberg said.

A transcript of the CBA testimony can be accessed on the CBA website.  

More comprehensive comments will be posted to the FDA docket by the deadline of Sept. 27.  CBA members who have additional points to suggest are encouraged to submit them through a comments page on the CBA website.