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. 2019 Sep;39(Suppl 1):20-30.
doi: 10.1038/s41372-019-0469-8.

Issues, challenges, and the way forward in conducting clinical trials among neonates: investigators' perspective

Sindhu Sivanandan  1 , Kajal Jain  2 , Nishad Plakkal  1 , Monika Bahl  3 , Tanushree Sahoo  2 , Shirshendu Mukherjee  4 , Yogendra Kumar Gupta  5 , Ramesh Agarwal  6
Affiliations

Issues, challenges, and the way forward in conducting clinical trials among neonates: investigators' perspective

Sindhu Sivanandan et al. J Perinatol. 2019 Sep.

Abstract

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.

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Conflict of interest statement

The authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1
Process of regulatory approval for clinical trials in India
Fig. 2
Fig. 2
Process of reporting serious adverse events (SAE) during a regulatory clinical trial in India. CDSCO: Central Drugs Standard Control Organization, DCGI: Drug Controller General of India
See this image and copyright information in PMC

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