Pulmonary hypertension in interstitial lung disease: Clinical trial design and endpoints: A consensus statement from the Pulmonary Vascular Research Institute's Innovative Drug Development Initiative—Group 3 Pulmonary Hypertension

• Published in: Pulmonary circulation Vol. 12; no. 4; pp. e12178 - n/a
• Main Authors: Nathan, Steven D., Fernandes, Peter, Psotka, Mitchell, Vitulo, Patrizio, Piccari, Lucilla, Antoniou, Katerina, Nikkho, Sylvia M., Stockbridge, Norman
• Format: Journal Article
• Language: English
• Published: United States John Wiley & Sons, Inc 01-10-2022; John Wiley and Sons Inc; Wiley
• Subjects: clinical trial design; Clinical trials; Connective tissue; Contraindications; Drug development; Genotype & phenotype; interstitial lung disease; Lung diseases; Nitric oxide; Pneumonia; Pulmonary fibrosis; Pulmonary hypertension; Review; interstitial lung disease; clinical trial design; pulmonary hypertension
• ISSN: 2045-8940; 2045-8932; 2045-8940
• DOI: 10.1002/pul2.12178

Pulmonary hypertension (PH) associated with interstitial lung disease (ILD) is an attractive target for clinical trials of PH medications. There are many factors that need to be considered to prime such studies for success. The patient phenotype most likely to respond to the intervention requires weighing the extent of the parenchymal lung disease against the severity of the hemodynamic impairment. The inclusion criteria should not be too restrictive, thus enabling recruitment. The trial should be of sufficient duration to meet the chosen endpoint which should reflect how the patient feels, functions, or survives. This paper summarizes prior studies in PH‐ILD and provides a framework of the type of studies to be considered. Inclusion criteria, clinical trial endpoints, and pharmacovigilance in the context of PH‐ILD trials are also addressed. Through lessons learnt from prior studies, suggestions and guidance for future clinical trials in PH‐ILD are also provided.