The purpose of this project is to select approvable drugs for the treatment of four orphan diseases: sickle cell disease, thalassemia, hemochromatosis, and erythropoietic porphyries. From the available world medical literature on the four diseases, drugs that are claimed to be effective will be evaluated and categorized as to the extent of clinical testing and animal experimentation. The therapeutic reviews will be examined by a group of consultants who will in turn select approvable drugs for submission to the Orphan Products Board. Promising drugs that need additional but minimal research will be selected for Phase 11 studies on animals and patients. It is expected that this project will hasten the selection process and commercial introduction of drugs for orphan diseases in general, particularly of hereditary erythrocytic diseases.National Heart, Lung and Blood Institute
