Clinical trial design and new therapies for pulmonary arterial hypertension

Olivier Sitbon, Mardi Gomberg-Maitland, John Granton, Michael I. Lewis, Stephen Mathai, Maurizio Rainisio, Norman L. Stockbridge, Martin R. Wilkins, Roham T. Zamanian, Lewis J. Rubin

Research output: Contribution to journalArticle

Abstract

Until 20 years ago the treatment of pulmonary arterial hypertension (PAH) was based on case reports and small series, and was largely ineffectual. As a deeper understanding of the pathogenesis and pathophysiology of PAH evolved over the subsequent two decades, coupled with epidemiological studies defining the clinical and demographic characteristics of the condition, a renewed interest in treatment development emerged through collaborations between international experts, industry and regulatory agencies. These efforts led to the performance of robust, high-quality clinical trials of novel therapies that targeted putative pathogenic pathways, leading to the approval of more than 10 novel therapies that have beneficially impacted both the quality and duration of life. However, our understanding of PAH remains incomplete and there is no cure. Accordingly, efforts are now focused on identifying novel pathogenic pathways that may be targeted, and applying more rigorous clinical trial designs to better define the efficacy of these new potential treatments and their role in the management scheme. This article, prepared by a Task Force comprised of expert clinicians, trialists and regulators, summarises the current state of the art, and provides insight into the opportunities and challenges for identifying and assessing the efficacy and safety of new treatments for this challenging condition.

Original languageEnglish (US)
JournalThe European respiratory journal
Volume53
Issue number1
DOIs
StatePublished - Jan 1 2019

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Pulmonary Hypertension
Clinical Trials
Advisory Committees
Epidemiologic Studies
Industry
Therapeutics
Quality of Life
Demography
Safety

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine

Cite this

Sitbon, O., Gomberg-Maitland, M., Granton, J., Lewis, M. I., Mathai, S., Rainisio, M., ... Rubin, L. J. (2019). Clinical trial design and new therapies for pulmonary arterial hypertension. The European respiratory journal, 53(1). https://doi.org/10.1183/13993003.01908-2018

Clinical trial design and new therapies for pulmonary arterial hypertension. / Sitbon, Olivier; Gomberg-Maitland, Mardi; Granton, John; Lewis, Michael I.; Mathai, Stephen; Rainisio, Maurizio; Stockbridge, Norman L.; Wilkins, Martin R.; Zamanian, Roham T.; Rubin, Lewis J.

In: The European respiratory journal, Vol. 53, No. 1, 01.01.2019.

Research output: Contribution to journalArticle

Sitbon, O, Gomberg-Maitland, M, Granton, J, Lewis, MI, Mathai, S, Rainisio, M, Stockbridge, NL, Wilkins, MR, Zamanian, RT & Rubin, LJ 2019, 'Clinical trial design and new therapies for pulmonary arterial hypertension', The European respiratory journal, vol. 53, no. 1. https://doi.org/10.1183/13993003.01908-2018
Sitbon, Olivier ; Gomberg-Maitland, Mardi ; Granton, John ; Lewis, Michael I. ; Mathai, Stephen ; Rainisio, Maurizio ; Stockbridge, Norman L. ; Wilkins, Martin R. ; Zamanian, Roham T. ; Rubin, Lewis J. / Clinical trial design and new therapies for pulmonary arterial hypertension. In: The European respiratory journal. 2019 ; Vol. 53, No. 1.
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