Real-world evidence to advance knowledge in pulmonary hypertension: Status, challenges, and opportunities. A consensus statement from the Pulmonary Vascular Research Institute's Innovative Drug Development Initiative's Real-world Evidence Working Group.

This manuscript on real-world evidence (RWE) in pulmonary hypertension (PH) incorporates the broad experience of members of the Pulmonary Vascular Research Institute's Innovative Drug Development Initiative Real-World Evidence Working Group. We aim to strengthen the research community's understanding of RWE in PH to facilitate clinical research advances and ultimately improve patient care. Herein, we review real-world data (RWD) sources, discuss challenges and opportunities when using RWD sources to study PH populations, and identify resources needed to support the generation of meaningful RWE for the global PH community.

Informations de copyright

© 2023 The Authors. Pulmonary Circulation published by John Wiley & Sons Ltd on behalf of Pulmonary Vascular Research Institute.

Déclaration de conflit d'intérêts

Boucly reports consulting fees from AOP Orphan; payment, honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from AOP Orphan, MSD, and Janssen; and support for attending meetings/travel from MSD and Janssen. Moles reports grants or contracts from Acceleron Pharma, Janssen, and CVS Caremark; and leadership or fiduciary role in other board, society, committee, or advocacy group, paid or unpaid, for Gossamer Bio. Elwing reports grants or contracts from United Therapeutics Corporation, Gossamer Bio, Bayer, Acceleron/Merck, Altavant, Aerovate, Tenax, Pharmosa, Actelion/Janssen, Arena, Lung, Liquidia, and Phase Bio; consulting fees for United Therapeutics Corporation; support for attending meetings and/or travel for United Therapeutics, Altavant, Aerovate, Bayer, Gossamer Bio, Liquida, Acceleron/Merck, Janssen/Actelion, and Insmed; and other financial or nonfinancial interests from Bayer as a grant reviewer. Jose reports grants or contracts from Team Phenomenal Hope Charity, Francis Family Foundation, United Therapeutics, and Janssen/Johnson & Johnson. Alves Jr. reports payment or honoraria from Bayer and Janssen; and support for attending meetings and/or travel from Janssen. Minty reports consulting fees from Janssen Pharmaceuticals and Orpyx Medical Technologies; and stock or stock options from Orpyx Medical Technologies. Weatherald reports grants or contracts from Janssen, Bayer, Merck, Canadian Institutes for Health Research, and the Heart & Stroke Foundation of Canada; consulting fees from Janssen and Merck; payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Janssen and Merck; payment for expert testimony from Sprigings Intellectual Property Law; support for attending meetings and/or travel from Janssen; participation on a data safety monitoring board or advisory board from Janssen, Acceleron, and Université Laval; and leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid, from the Pulmonary Hypertension Association of Canada. Mathai reports grants or contracts from NIH/NHLBI (HL125175) and DOD (W81XWH‐20‐1‐0768); consulting fees from Actelion/Janssen, Aerovate, Merck, United Therapeutics Corporation, and Theravance; and participation on a data safety monitoring board or advisory board from Bayer. Sahay reports grants or contracts from United Therapeutics Corporation, Janssen, Bayer, Merck, Liquidia, and Gossamer; consulting fees from Merck, Liquidia, and Gossamer; payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from United Therapeutics Corporation (received by hospital rather than self); patents planned, issued, or pending with Janssen; and participation on a data safety monitoring board or advisory board with GSK, Bayer, United Therapeutics, and Janssen. Pulido reports grants or contracts from Janssen, MSD, and United Therapeutics; consulting fees from Janssen, Bayer, Gossamer, and MSD; and payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Bayer, Janssen, and Pfizer. Badagliacca reports grants or contracts from United Therapeutics Corporation, Ferrer, and Janssen; consulting fees from MSD, United Therapeutics Corporation, Ferrer, Janssen, and AOP; payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from MSD, United Therapeutics Corporation, Ferrer, Janssen, and AOP; and support for attending meetings and/or travel from MSD, United Therapeutics Corporation, Ferrer, Janssen, and AOP. Cirulis reports grants or contracts from Entelligence (now ATS); and leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid, with ATS Web Committee Co‐Director and ATS Program Committee. Gerges reports grants or contracts from AOP Health; payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from AOP Health, Actelion/Janssen, and Ferrer; and support for attending meetings and/or travel from AOP Health, Actelion/Janssen, Ferrer, Daiichi Sankyo, and MSD. Gillmeyer reports grants or contracts from Parker B. Francis Fellowship Award, Doris Duke Charitable Foundation Award, and National Center for Advancing Translational Sciences, National Institutes of Health (BU‐CTSI Grant Number 1UL1TR001430); and leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid, for the American Thoracic Society Program Committee (unpaid). Hobohm reports consulting fees for Janssen, Boston Consulting, and MSD. Lau reports grants or contracts from Janssen and GSK; consulting fees for Janssen; and payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Janssen. Morland reports grants or contracts from United Therapeutics Corporation; leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid, from the PVRI Innovative Drug Development Initiative (unpaid); and stock or stock options from United Therapeutics Corporation. Nikkho reports grants or contracts from Bayer AG; and leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid, from the PVRI Innovative Drug Development Initiative (unpaid). The remaining authors declare no conflict of interest.