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Statement Before CTGTAC on “Draft Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products”

A Statement Before the Cellular, Tissue and Gene Therapies Advisory Committee
26 February 2014

"Draft Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products"

Rafael Cassata, MS, RAC (US, EU)
Deputy Director, Regulatory Affairs, Center for Cellular Therapies, AABB

AABB is an international, not-for-profit association representing individuals and institutions involved in the field of transfusion medicine and cellular therapies. The association is committed to improving health by developing and delivering standards, accreditation and educational programs that focus on optimizing patient and donor care and safety. AABB membership consists of nearly 2,000 institutions and 8,000 individuals, including physicians, nurses, scientists, researchers, administrators, medical technologists and other health care providers. AABB members are located in more than 80 countries.

We would like to thank the Food and Drug Administration (FDA) for bringing this topic for discussion to the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC). We would also like to commend FDA for developing this draft guidance, which, when finalized, will serve as an instrumental tool in contributing to the design of many phase 1 clinical trials of cell and gene therapy products.

AABB has developed preliminary comments to the draft guidance document, which will be submitted to the docket before the end of the comment period. In general, we think that the specific sections (Sections IV.B an IV.F) highlighted for today's discussion are of the most concern for AABB's membership. Specifically, we would like to iterate a few issues relevant to today's discussion:

  • We recommend including "Geriatrics" to the list of study population groups listed in Section IV.B. Geriatrics often have co-morbidities and concomitant therapies that may interact with the investigational cell and gene therapy product. These age-associated issues may not be observed or extrapolated from the adult population.
  • In Section IV.F.3 (Monitoring and Follow-up, Duration of Follow-up), FDA recommends that long-term monitoring not be designed as a separate protocol. However, for academic centers or any other organizations, federal funding will not allow carryover grant money to cover long-term follow-up. Additionally, there are logistical challenges with keeping a database open, especially, if the clinical development of the cell or gene therapy product will progress to phase 2 and 3 trials.

Thank you for this opportunity and we will submit our complete set of comments before the end of the comment period.