The Creating Hope Act: what is old is new again

Abstract

Jaszianne A Tolbert,1,2 Jennifer L Goldman,2-4 Ralph E Kauffman,2,4 Susan M Abdel-Rahman2,41Division of Hematology/Oncology, 2Division of Clinical Pharmacology and Therapeutic Innovation, 3Division of Infectious Diseases, Children's Mercy Hospital, Kansas City, MO, USA; 4Department of Pediatrics, University of Missouri-Kansas City, School of Medicine, Kansas City, MO, USAAbstract: The Creating Hope Act, passed as part of the Food and Drug Administration Safety and Innovation Act of 2012, is among the newest laws intended to foster drug development for rare and neglected diseases in children. The act expands the priority review voucher incentive that first appeared in the Food and Drug Administration Amendments Act of 2007 and was intended to stimulate the development of products for the prevention and treatment of tropical diseases. Notably, legislative and regulatory initiatives aimed at enhancing drug development both for use in children and for rare diseases have intermittently emerged over the past 3 decades. This manuscript provides an overview of related legislation that has preceded the Creating Hope Act and examines the potential impact of the new act in the context of the outcomes that have been observed with the earlier initiatives.Keywords: orphan drug, rare disease, pediatric, drug development, priority review voucher