J Am Coll Cardiol. 2017 Jul 25;70(4):495-503. doi: 10.1016/j.jacc.2017.04.007. Epub 2017 Jun 29.
The STARTS-1 and -2 trials (Sildenafil in Treatment-Naive Children, Aged 1 to 17 Years, With Pulmonary Arterial Hypertension) and subsequent 2012 U.S. Food and Drug Administration (FDA) product labeling for sildenafil use in pediatric patients with pulmonary hypertension highlight many of the challenges to the development and approval of medications for children. This experience served as the impetus for direct collaboration between FDA representatives and the Joint Council on Congenital Heart Disease (JCCHD) (representing the pediatric cardiology leadership of the American College of Cardiology, the American Heart Association, and the American Academy of Pediatrics) to improve communication and realign missions with regard to pediatric drug trials. These discussions led to the joint FDA/JCCHD development of this statement, which describes the current environment and identifies possible future directions for reducing barriers to pediatric drug trials.
STARTS-1 和 -2 试验（治疗初治儿童肺动脉高压的西地那非，年龄 1 至 17 岁）以及随后的 2012 年美国食品和药物管理局（FDA）关于西地那非在儿科肺动脉高压患者中的使用的产品标签突出了许多开发和批准儿童用药的挑战。这一经验促使 FDA 代表与先天性心脏病联合委员会（JCCHD）（代表美国心脏病学会、美国心脏协会和美国儿科学会的儿科心脏病学领导层）直接合作，以改善沟通并重新调整儿科药物试验的任务。这些讨论导致了 FDA/JCCHD 的联合制定本声明，该声明描述了当前的环境，并确定了减少儿科药物试验障碍的可能未来方向。
