Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension

This manuscript on endpoints incorporates the broad experience of members of Pulmonary Vascular Research Institute’s Innovative Drug Development Initiative as an open debate platform for academia, the pharmaceutical industry and regulatory experts surrounding the future design of clinical trials in...

Descripción completa

Detalles Bibliográficos
Autores principales: Sitbon, Olivier, Nikkho, Sylvia, Benza, Raymond, (CQ) Deng, Chunqin, Farber, Harrison W., Gomberg-Maitland, Mardi, Hassoun, Paul, Meier, Christian, Pepke-Zaba, Joanna, Prasad, Krishna, Seeger, Werner, Corris, Paul A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2020
Materias:
Consensus Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7682238/
https://www.ncbi.nlm.nih.gov/pubmed/33282190
http://dx.doi.org/10.1177/2045894020962960
_version_ 1783612672701366272
author Sitbon, Olivier
Nikkho, Sylvia
Benza, Raymond
(CQ) Deng, Chunqin
Farber, Harrison W.
Gomberg-Maitland, Mardi
Hassoun, Paul
Meier, Christian
Pepke-Zaba, Joanna
Prasad, Krishna
Seeger, Werner
Corris, Paul A
author_facet Sitbon, Olivier
Nikkho, Sylvia
Benza, Raymond
(CQ) Deng, Chunqin
Farber, Harrison W.
Gomberg-Maitland, Mardi
Hassoun, Paul
Meier, Christian
Pepke-Zaba, Joanna
Prasad, Krishna
Seeger, Werner
Corris, Paul A
author_sort Sitbon, Olivier
collection PubMed
description This manuscript on endpoints incorporates the broad experience of members of Pulmonary Vascular Research Institute’s Innovative Drug Development Initiative as an open debate platform for academia, the pharmaceutical industry and regulatory experts surrounding the future design of clinical trials in pulmonary hypertension. It reviews our current understanding of endpoints used in phase 2 and 3 trials for pulmonary hypertension and discusses in detail the value of newer approaches. These include the roles of composite endpoints and how these can be developed and validated. The newer concept of risk analysis is also discussed, including how such risk scores might be utilised as endpoints in clinical trials.
format Online
Article
Text
id pubmed-7682238
institution National Center for Biotechnology Information
language English
publishDate 2020
publisher SAGE Publications
record_format MEDLINE/PubMed
spelling pubmed-76822382020-12-03 Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension Sitbon, Olivier Nikkho, Sylvia Benza, Raymond (CQ) Deng, Chunqin Farber, Harrison W. Gomberg-Maitland, Mardi Hassoun, Paul Meier, Christian Pepke-Zaba, Joanna Prasad, Krishna Seeger, Werner Corris, Paul A Pulm Circ Consensus Report This manuscript on endpoints incorporates the broad experience of members of Pulmonary Vascular Research Institute’s Innovative Drug Development Initiative as an open debate platform for academia, the pharmaceutical industry and regulatory experts surrounding the future design of clinical trials in pulmonary hypertension. It reviews our current understanding of endpoints used in phase 2 and 3 trials for pulmonary hypertension and discusses in detail the value of newer approaches. These include the roles of composite endpoints and how these can be developed and validated. The newer concept of risk analysis is also discussed, including how such risk scores might be utilised as endpoints in clinical trials. SAGE Publications 2020-11-18 /pmc/articles/PMC7682238/ /pubmed/33282190 http://dx.doi.org/10.1177/2045894020962960 Text en © The Author(s) 2020 https://creativecommons.org/licenses/by/4.0/ Creative Commons CC-BY: This article is distributed under the terms of the Creative Commons Attribution 4.0 License (https://creativecommons.org/licenses/by/4.0/) which permits any use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage).
spellingShingle Consensus Report
Sitbon, Olivier
Nikkho, Sylvia
Benza, Raymond
(CQ) Deng, Chunqin
Farber, Harrison W.
Gomberg-Maitland, Mardi
Hassoun, Paul
Meier, Christian
Pepke-Zaba, Joanna
Prasad, Krishna
Seeger, Werner
Corris, Paul A
Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
title Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
title_full Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
title_fullStr Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
title_full_unstemmed Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
title_short Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
title_sort novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
topic Consensus Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7682238/
https://www.ncbi.nlm.nih.gov/pubmed/33282190
http://dx.doi.org/10.1177/2045894020962960
work_keys_str_mv AT sitbonolivier novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT nikkhosylvia novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT benzaraymond novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT cqdengchunqin novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT farberharrisonw novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT gombergmaitlandmardi novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT hassounpaul novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT meierchristian novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT pepkezabajoanna novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT prasadkrishna novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT seegerwerner novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension
AT corrispaula novelcompositeclinicalendpointsandriskscoresusedinclinicaltrialsinpulmonaryarterialhypertension

Ejemplares similares