Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER), Puducherry, India.
All India Institute of Medical Sciences (AIIMS), New Delhi, India.
J Perinatol. 2019 Sep;39(Suppl 1):20-30. doi: 10.1038/s41372-019-0469-8.
Clinical trials are essential to test the safety and efficacy of new treatments in any population. The paucity of drug trials especially in the neonatal population has led to the widespread use of unlicensed or off-label medications, exposing them to the risks of drug toxicity and ineffective treatment. Ethical and operational challenges are no longer considered valid excuses for not conducting drug trials in neonates. We recently participated in a combined phase-2 and phase-3 trial investigating a new indigenous goat lung surfactant extract (GLSE) for the treatment of respiratory distress syndrome (RDS) in preterm neonates. In this article, we share pertinent challenges faced by us during the trial to better inform and foster-positive discussion among drug developers, administrators, regulatory authorities, patient advocacy groups, and researchers. Also, we provide many tools developed for the GLSE trial that can be modified and used by prospective trialists.
临床试验对于在任何人群中测试新治疗方法的安全性和有效性至关重要。药物试验特别是在新生儿人群中的缺乏，导致了未经许可或超适应证用药的广泛使用，使他们面临药物毒性和治疗无效的风险。伦理和操作方面的挑战不再被视为在新生儿中进行药物试验的合理借口。我们最近参与了一项联合的 2 期和 3 期试验，研究一种新的本土山羊肺表面活性剂提取物（GLSE）治疗早产儿呼吸窘迫综合征（RDS）。在本文中，我们分享了在试验中遇到的相关挑战，以便在药物开发者、管理者、监管机构、患者权益团体和研究人员之间更好地进行信息交流和促进积极讨论。此外，我们还提供了为 GLSE 试验开发的许多工具，这些工具可以为未来的试验人员进行修改和使用。
