Food and Drug Administration's Proposed Approach to Regulation of Hematopoietic Stem/Progenitor Cell Products for Therapeutic use

At present, FDA regulatory oversight of hematopoietic stem/progenitor cells occurs when the cells, regardless of their source (marrow, peripheral, or placental/umbilical cord blood), are manipulated in a manner that may alter their biological function (eg, ex vivo expansion, genetic modification). Such cells are considered as somatic cell or gene therapy products and are reviewed by the FDA through the IND application process. When cellular products are processed with devices that require premarket approval, the devices are reviewed by the FDA through an Investigational Device Exemption application. Minimally manipulated hematopoietic stem/progenitor cells (processed such that the biological function is not altered) are currently not regulated by the FDA (Table 3). The agency's proposed regulatory approach pertains to hematopoietic stem/progenitor cells obtained from peripheral or placental/umbilical cord blood and does not encompass bone marrow-derived cells (Table 3). Unrelated allogeneic bone marrow federal oversight currently exists through the Health Resources and Services Administration contract with the National Marrow Donor Program. The current approach proposes that all manufacturers of peripheral or placental/umbilical cord blood-derived hematopoietic stem/progenitor cell products intended for human transplantation or infusion register and list their products with FDA, follow applicable donor suitability determination processes, and apply good tissue practices to be set forth by the FDA. Donor suitability determination would be required for all allogeneic donors and recommended for autologous donors. The FDA proposes that unrelated allogeneic products would become licensed products when process and establishment controls and product standards have been adequately developed. The proposed approach to develop such product standards represents a novel FDA attempt to develop such standards from existing data in cooperation with public effort.