Mokshagundam Deepa, Rosenthal David N, Smyth Lauren, Lorts Angela
St Louis Children's Hospital, Washington University School of Medicine, St Louis, Missouri.
Lucile Packard Children's Hospital Stanford, Stanford University School of Medicine, Palo Alto, California.
J Heart Lung Transplant. 2025 Apr;44(4):541-544. doi: 10.1016/j.healun.2024.12.018.
Progress in the field of pediatric ventricular assist devices (VAD) have revolutionized the care and management of young patients with end-stage heart failure. However, innovation in pediatric medical devices is limited by financial and regulatory barriers due to the small and heterogenous target population. The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) was established in 2017 as a learning network to improve outcomes of pediatric patients supported on VAD. Here we describe how intentional design of the ACTION registry has mitigated regulatory and financial barriers to pediatric VAD innovation through the use of real-world evidence gathered through a learning network.
小儿心室辅助装置（VAD）领域的进展彻底改变了终末期心力衰竭年轻患者的护理和管理方式。然而，由于目标人群规模小且异质性大，儿科医疗设备的创新受到资金和监管障碍的限制。高级心脏治疗改善结局网络（ACTION）于2017年成立，作为一个学习网络，旨在改善接受VAD支持的儿科患者的结局。在此，我们描述了ACTION注册研究的精心设计如何通过利用通过学习网络收集的真实世界证据，减轻了儿科VAD创新的监管和资金障碍。
