Chronic pulmonary and respiratory conditions associated with preterm birth are incompletely characterized, complicating long-term treatment and development of more effective therapies. Stakeholders face challenges in the development of validated, clinically meaningful endpoints that adequately measure morbidities and predict or represent health outcomes for preterm neonates. We propose in this paper a research agenda, informed by the input of experts from a 2018 workshop we convened on this topic, to advance endpoint and treatment development. We discuss the necessity of further evaluation of existing endpoints and the improved characterization of disease endotypes. We also discuss key steps to the development of optimized short- and long-term endpoints that can be linked to meaningful health outcomes. Finally, we discuss the importance of limiting variability in data collection and the application of new clinical trial endpoints as well as the critical nature of multi-stakeholder collaboration to advancing therapeutic development for this vulnerable patient population.
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The development of this manuscript was supported by the U.S. Food and Drug Administration (7U19FD004971-04). This manuscript reflects the views of the authors and should not be construed to represent FDA’s views or policies.
Conflicts of interest
Dr. Daniel receives Consulting Fees from AbbVie, Reagan-Udall Foundation, and Genentech and is the Editor-in-Chief of TIRS. The authors have no other conflicts of interest to declare.
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Sheehan, S., Baer, G., Romine, M. et al. Advancing Therapeutic Development for Pulmonary Morbidities Associated with Preterm Birth. Ther Innov Regul Sci 54, 1312–1318 (2020). https://doi.org/10.1007/s43441-020-00153-z
- Bronchopulmonary dysplasia
- Chronic pulmonary insufficiency of prematurity
- Endpoint development
- Pulmonary outcomes