Collaboration in Regulatory Science to Facilitate Therapeutic Development for Neonates

Title:Collaboration in Regulatory Science to Facilitate Therapeutic Development for Neonates

Volume: 23 Issue: 38

Author(s): Susan K. McCune*, Yeruk Ager Mulugeta and Gerri R. Baer

Affiliation:

• Office of Pediatric Therapeutics, Office of the Commissioner, Food and Drug Administration, Silver Spring, MD,United States

Keywords: Suggested keywords are neonate, drug label, regulatory science, innovative trial design, consortia, research networks.

Abstract: Background: Historically, neonatal therapeutic interventions were derived from adult therapeutics, and tragedies resulting from this approach have demonstrated differences in the pathophysiologic and developmental processes between neonates and older patients. Over the past 3 decades, researchers and collaborative research networks have made progress in the systematic evaluation of neonatal therapies, yet most neonatal therapeutic products have been incompletely assessed for safety and efficacy, and remain unlabeled and unapproved.

Approach: This work describes the legislative initiatives that have stimulated an increase in pediatric and neonatal studies. It highlights examples of successful neonatal drug studies that have resulted in informative neonatal labeling changes, as well as studies that have produced incomplete information. Strategies that support the design of successful studies, including targeting specific subpopulations, modeling and simulation to inform dose selection, innovative design strategies, biomarkers, and endpoints are discussed. Multi-stakeholder consortia such as the International Neonatal Consortium (INC), are working to improve the tools needed for the development of neonatal therapies. These research tools may be used by trial networks to inform consistent and efficient multicenter studies.

Conclusion: More data are needed to support safe and effective use of drugs in neonates, and to obtain these data, a thorough understanding of pathophysiology, drug disposition, biomarkers, and clinically-meaningful endpoints is required. This information will be derived from clinical trials, registries, real-world evidence, and the medical literature. Collaboration of consortia and the development of research networks are essential to achieving these goals.

Cite this article as:

McCune K. Susan *, Mulugeta Ager Yeruk and Baer R. Gerri, Collaboration in Regulatory Science to Facilitate Therapeutic Development for Neonates, Current Pharmaceutical Design 2017; 23 (38) . https://dx.doi.org/10.2174/1381612823666170926114857

DOI https://dx.doi.org/10.2174/1381612823666170926114857	Print ISSN 1381-6128
Publisher Name Bentham Science Publisher	Online ISSN 1873-4286