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Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data

The 21(st) Century Cures Act requires FDA to expand its use of real-world evidence (RWE) to support approval of previously approved drugs for new disease indications and post-marketing study requirements. To address this need in neonates, the FDA and the Critical Path Institute (C-Path) established...

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Autores principales: Barrett, Jeffrey S., Cala Pane, Megan, Knab, Timothy, Roddy, William, Beusmans, Jack, Jordie, Eric, Singh, Kanwaljit, Davis, Jonathan Michael, Romero, Klaus, Padula, Michael, Thebaud, Bernard, Turner, Mark
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9597633/
https://www.ncbi.nlm.nih.gov/pubmed/36313352
http://dx.doi.org/10.3389/fphar.2022.988974
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author Barrett, Jeffrey S.
Cala Pane, Megan
Knab, Timothy
Roddy, William
Beusmans, Jack
Jordie, Eric
Singh, Kanwaljit
Davis, Jonathan Michael
Romero, Klaus
Padula, Michael
Thebaud, Bernard
Turner, Mark
author_facet Barrett, Jeffrey S.
Cala Pane, Megan
Knab, Timothy
Roddy, William
Beusmans, Jack
Jordie, Eric
Singh, Kanwaljit
Davis, Jonathan Michael
Romero, Klaus
Padula, Michael
Thebaud, Bernard
Turner, Mark
author_sort Barrett, Jeffrey S.
collection PubMed
description The 21(st) Century Cures Act requires FDA to expand its use of real-world evidence (RWE) to support approval of previously approved drugs for new disease indications and post-marketing study requirements. To address this need in neonates, the FDA and the Critical Path Institute (C-Path) established the International Neonatal Consortium (INC) to advance regulatory science and expedite neonatal drug development. FDA recently provided funding for INC to generate RWE to support regulatory decision making in neonatal drug development. One study is focused on developing a validated definition of bronchopulmonary dysplasia (BPD) in neonates. BPD is difficult to diagnose with diverse disease trajectories and few viable treatment options. Despite intense research efforts, limited understanding of the underlying disease pathobiology and disease projection continues in the context of a computable phenotype. It will be important to determine if: 1) a large, multisource aggregation of real-world data (RWD) will allow identification of validated risk factors and surrogate endpoints for BPD, and 2) the inclusion of these simulations will identify risk factors and surrogate endpoints for studies to prevent or treat BPD and its related long-term complications. The overall goal is to develop qualified, fit-for-purpose disease progression models which facilitate credible trial simulations while quantitatively capturing mechanistic relationships relevant for disease progression and the development of future treatments. The extent to which neonatal RWD can inform these models is unknown and its appropriateness cannot be guaranteed. A component of this approach is the critical evaluation of the various RWD sources for context-of use (COU)-driven models. The present manuscript defines a landscape of the data including targeted literature searches and solicitation of neonatal RWD sources from international stakeholders; analysis plans to develop a family of models of BPD in neonates, leveraging previous clinical trial experience and real-world patient data is also described.
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spelling pubmed-95976332022-10-27 Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data Barrett, Jeffrey S. Cala Pane, Megan Knab, Timothy Roddy, William Beusmans, Jack Jordie, Eric Singh, Kanwaljit Davis, Jonathan Michael Romero, Klaus Padula, Michael Thebaud, Bernard Turner, Mark Front Pharmacol Pharmacology The 21(st) Century Cures Act requires FDA to expand its use of real-world evidence (RWE) to support approval of previously approved drugs for new disease indications and post-marketing study requirements. To address this need in neonates, the FDA and the Critical Path Institute (C-Path) established the International Neonatal Consortium (INC) to advance regulatory science and expedite neonatal drug development. FDA recently provided funding for INC to generate RWE to support regulatory decision making in neonatal drug development. One study is focused on developing a validated definition of bronchopulmonary dysplasia (BPD) in neonates. BPD is difficult to diagnose with diverse disease trajectories and few viable treatment options. Despite intense research efforts, limited understanding of the underlying disease pathobiology and disease projection continues in the context of a computable phenotype. It will be important to determine if: 1) a large, multisource aggregation of real-world data (RWD) will allow identification of validated risk factors and surrogate endpoints for BPD, and 2) the inclusion of these simulations will identify risk factors and surrogate endpoints for studies to prevent or treat BPD and its related long-term complications. The overall goal is to develop qualified, fit-for-purpose disease progression models which facilitate credible trial simulations while quantitatively capturing mechanistic relationships relevant for disease progression and the development of future treatments. The extent to which neonatal RWD can inform these models is unknown and its appropriateness cannot be guaranteed. A component of this approach is the critical evaluation of the various RWD sources for context-of use (COU)-driven models. The present manuscript defines a landscape of the data including targeted literature searches and solicitation of neonatal RWD sources from international stakeholders; analysis plans to develop a family of models of BPD in neonates, leveraging previous clinical trial experience and real-world patient data is also described. Frontiers Media S.A. 2022-10-12 /pmc/articles/PMC9597633/ /pubmed/36313352 http://dx.doi.org/10.3389/fphar.2022.988974 Text en Copyright © 2022 Barrett, Cala Pane, Knab, Roddy, Beusmans, Jordie, Singh, Davis, Romero, Padula, Thebaud and Turner. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Pharmacology
Barrett, Jeffrey S.
Cala Pane, Megan
Knab, Timothy
Roddy, William
Beusmans, Jack
Jordie, Eric
Singh, Kanwaljit
Davis, Jonathan Michael
Romero, Klaus
Padula, Michael
Thebaud, Bernard
Turner, Mark
Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data
title Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data
title_full Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data
title_fullStr Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data
title_full_unstemmed Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data
title_short Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data
title_sort landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: leveraging clinical trial experience and real-world data
topic Pharmacology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9597633/
https://www.ncbi.nlm.nih.gov/pubmed/36313352
http://dx.doi.org/10.3389/fphar.2022.988974
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