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

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...

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Main Authors: Olivier Sitbon, Sylvia Nikkho, Raymond Benza, Chunqin (CQ) Deng, Harrison W. Farber, Mardi Gomberg-Maitland, Paul Hassoun, Christian Meier, Joanna Pepke-Zaba, Krishna Prasad, Werner Seeger, Paul A Corris
Format: Article
Language:English
Published: SAGE Publishing 2020-11-01
Series:Pulmonary Circulation
Online Access:https://doi.org/10.1177/2045894020962960
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spelling doaj-f01c651180584100ab272cef141d8f922020-12-01T06:03:24ZengSAGE PublishingPulmonary Circulation2045-89402020-11-011010.1177/2045894020962960Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertensionOlivier Sitbon0Sylvia Nikkho1Raymond Benza2Chunqin (CQ) Deng3Harrison W. Farber4Mardi Gomberg-Maitland5Paul Hassoun6Christian Meier7Joanna Pepke-Zaba8Krishna Prasad9Werner Seeger10Paul A Corris11Assistance Publique–Hôpitaux de Paris, Service de Pneumologie, Hôpital Bicêtre, Université Paris-Sud, Laboratoire d’Excellence en Recherche sur le Médicament et Innovation Thérapeutique, Le Kremlin-Bicêtre, France, Clinical Development, Berlin, GermanyThe Ohio State University, Columbus, OH, USAoration, Raleigh-Durham, NC, USATufts Medical Center, Boston, MA, USA of Medicine and Health Sciences, Washington, DC, USA, Baltimore, MD, USA, Clinical Development, Berlin, GermanyRoyal , Cambridge, UKMedicines and Healthcare Products Regulatory Agency (MHRA), London, UKUniversity of Giessen and Marburg Lung Center (UGMLC), Giessen, GermanyClinical and Translational Research Institute Newcastle University, Newcastle upon Tyne, UKThis 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.https://doi.org/10.1177/2045894020962960
collection DOAJ
language English
format Article
sources DOAJ
author Olivier Sitbon
Sylvia Nikkho
Raymond Benza
Chunqin (CQ) Deng
Harrison W. Farber
Mardi Gomberg-Maitland
Paul Hassoun
Christian Meier
Joanna Pepke-Zaba
Krishna Prasad
Werner Seeger
Paul A Corris
spellingShingle Olivier Sitbon
Sylvia Nikkho
Raymond Benza
Chunqin (CQ) Deng
Harrison W. Farber
Mardi Gomberg-Maitland
Paul Hassoun
Christian Meier
Joanna Pepke-Zaba
Krishna Prasad
Werner Seeger
Paul A Corris
Novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
Pulmonary Circulation
author_facet Olivier Sitbon
Sylvia Nikkho
Raymond Benza
Chunqin (CQ) Deng
Harrison W. Farber
Mardi Gomberg-Maitland
Paul Hassoun
Christian Meier
Joanna Pepke-Zaba
Krishna Prasad
Werner Seeger
Paul A Corris
author_sort Olivier Sitbon
title 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_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_sort novel composite clinical endpoints and risk scores used in clinical trials in pulmonary arterial hypertension
publisher SAGE Publishing
series Pulmonary Circulation
issn 2045-8940
publishDate 2020-11-01
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.
url https://doi.org/10.1177/2045894020962960
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