Obtaining an accepted Investigational New Drug application to operate an umbilical cord blood bank

BACKGROUND: The residual blood left in the placenta, previously considered a biologic waste, contains sufficient hematopoietic stem and progenitor cel ls to consistently engraft at least a small recipient. Over the past severa l years, more than 500 HLA-matched, related and unrelated, allogeneic cord blood transplants have been performed. Consequently, public and private cor d blood banks are being developed to meet future demands. Thus, the definit ion of a suitable and effective cord blood component needs to be critically defined. In February 1997, the US Food and Drug Administration (FDA) propo sed that cord blood banks should operate under an Investigational New Drug (IND) license. STUDY DESIGN AND METHODS: Standard operating procedures were designed using standards from the Foundation for Accreditation of Hematopoietic and Cellu lar Therapy, the American Association of Blood Banks, and the National Marr ow Donor Program and in accordance with current good manufacturing practice s. The standard operating procedures were field-tested and submitted to the FDA. RESULTS: Issues of the utmost concern to the FDA dealt with transplant reci pient outcome data collection, donor recruitment, sample tracking, the use of unlicensed materials, and the reporting of positive infectious disease r esults. After three attempts, an IND application was approved. CONCLUSIONS: To obtain approval of an IND application, cord blood banks nee d a set of standard operating procedures that describe cord blood collectio n, processing, freezing, and storage. Issues relating to potential cord blo od recipient identification, cord blood shipping, and reporting of transpla nt recipient outcomes are also needed. The IND process provides an opportun ity for outside reviewers to make suggestions that may be included in the s tandard operating procedures.