Abstract

There are many challenges to the development and approval of medications for children. The STARTS-1 and -2 trials, which are described briefly in this document, highlight many of the challenges in pediatric trial design and approval of medications for children, especially those with rare diseases. Through a series of conversations between the US Food and Drug Administration (FDA) and the Joint Council on Congenital Heart Disease (JCCHD) which had their origins around review of the effect of the STARTS-1 and -2 trials and subsequent regulatory response on clinical practice, it became clear that better communication and alignment of goals surrounding pediatric drug trials were needed. This paper serves as unique opportunity to bring leaders of the pediatric cardiology community and FDA together as one voice.