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 language | English (US) |
|---|---|
| Journal | The European respiratory journal |
| Volume | 53 |
| Issue number | 1 |
| DOIs | |
| State | Published - Jan 1 2019 |
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ASJC Scopus subject areas
- Pulmonary and Respiratory Medicine
Cite this
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 journal › Article
}
TY - JOUR
T1 - Clinical trial design and new therapies for pulmonary arterial hypertension
AU - Sitbon, Olivier
AU - Gomberg-Maitland, Mardi
AU - Granton, John
AU - Lewis, Michael I.
AU - Mathai, Stephen
AU - Rainisio, Maurizio
AU - Stockbridge, Norman L.
AU - Wilkins, Martin R.
AU - Zamanian, Roham T.
AU - Rubin, Lewis J.
PY - 2019/1/1
Y1 - 2019/1/1
N2 - 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.
AB - 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.
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U2 - 10.1183/13993003.01908-2018
DO - 10.1183/13993003.01908-2018
M3 - Article
C2 - 30545975
AN - SCOPUS:85060534130
VL - 53
JO - European Respiratory Journal, Supplement
JF - European Respiratory Journal, Supplement
SN - 0903-1936
IS - 1
ER -