Whereas the Office of Cellular and Gene Therapies (OCGT) of the FDA is responsible for regulatory oversight of therapies which may cure bleeding disorders;
Whereas the OCGT, a part of the Center for Biologics Evaluation and Review (CBER), has undergone significant reorganization as projects and resources have been shifted to the Center for Drug Evaluation and Review (CDER);
Whereas sufficient expertise and human resources are essential to safely move emerging technologies to potentially cure bleeding disorders into human clinical testing;
Therefore, the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation recommends that sufficient resources be allotted to OCGT to allow for
1. A collaborative relationship to accelerate the development of innovative clinical studies, and
2. Rapid review of translational research from academic institutions and industry.
