Abstract
Pulmonary hypertension (PH) and its subset, pulmonary arterial hypertension (PAH), are rare diseases with a significant unmet need. Between the 1980s and 2010s, the 5-year survival rate for PAH after diagnosis improved from 34% to 65%,12 but remains unacceptably low. Since the introduction of vasodilator therapy, 34 important advances have been made in the understanding of the disease pathophysiology and development of targeted therapies. There are now 14 US Food and Drug Administration (FDA)-approved therapies that target 3 distinct pathways that contribute to PAH, and additional therapeutic targets are currently under investigation in phase 1, 2, and 3 clinical trials.5 However, there have been major challenges in PH medication development to date, including: 1) only one medication approved for pediatric PAH; 2) focusing on vasodilator therapy rather than targeting the underlying pathogenesis of the disease; 3) no medications approved for PH World Health Organization (WHO) Groups 2, 3, and 5; and 4) several recent high-profile clinical failures after promising preclinical studies.The focus and goal of the PH research community should be directed at identifying new options and solutions for patients. The field must ensure that the approaches used for clinical trials to develop orphan drugs maximize the scarce resources available for recruiting subjects, and are directed toward making safe and effective therapies available in a timely manner. Therefore, there is a critical need to coordinate and harmonize innovative approaches within the field, including strengthening translational research to deliver promising candidates and optimize the designs, endpoints, and biomarkers to conduct safe and efficient clinical trials.
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