Congresswoman Yvette D. Clarke released the following statement on letters she sent to senior members of the Appropriations Committee to support the continuation of the Rare Pediatric Disease Priority Review program, and the inclusion of sickle cell disease in the program.
The program provides for the expedited review by the Food and Drug Administration of prescription drugs for disease that afflict fewer than 200,000 children in the United States. Almost 100,000 people in the United States, most of them African American, have sickle cell disease, which often results in serious infections and severe pain, and increases the risk of stroke.
"We desperately need to expand the medical treatments available for rare pediatric diseases, particularly sickle cell disease that afflicts many African Americans. This program has been a success, allowing pharmaceutical companies to introduce effective forms of medical treatment much faster than would otherwise have occurred. We have a shared responsibility to the families and children who need these health care choices. The incentives offered under the Rare Pediatric Disease Priority Review program will support the critical research on these diseases. With the inclusion of sickle cell disease in the program, we will offer hope to the many individuals who have the disease or who are concerned that their children will have it."