Advancing Therapeutic Development for Pulmonary Morbidities Associated with Preterm Birth.
Bronchopulmonary dysplasia
Chronic pulmonary insufficiency of prematurity
Endpoint development
Preterm
Pulmonary outcomes
Journal
Therapeutic innovation & regulatory science
ISSN: 2168-4804
Titre abrégé: Ther Innov Regul Sci
Pays: Switzerland
ID NLM: 101597411
Informations de publication
Date de publication:
11 2020
11 2020
Historique:
received:
13
02
2020
accepted:
10
04
2020
entrez:
1
12
2020
pubmed:
2
12
2020
medline:
4
6
2021
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 33258093
doi: 10.1007/s43441-020-00153-z
pii: 10.1007/s43441-020-00153-z
pmc: PMC9351457
mid: NIHMS1606457
doi:
Types de publication
Journal Article
Research Support, U.S. Gov't, Non-P.H.S.
Langues
eng
Sous-ensembles de citation
IM
Pagination
1312-1318Subventions
Organisme : Intramural FDA HHS
ID : FD999999
Pays : United States
Organisme : FDA HHS
ID : U19 FD004971
Pays : United States
Organisme : U.S. Food and Drug Administration
ID : 7U19FD004971-04
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