Clinical trial design, end-points, and emerging therapies in pulmonary arterial hypertension.

ABSTRACT: Clinical trials in pulmonary arterial hypertension (PAH) have led to the approval of several effective treatments that improve symptoms, exercise capacity and clinical outcomes. In phase 3 clinical trials, primary end-points must reflect how a patient "feels, functions or survives". In a rare disease like PAH, with an ever-growing number of treatment options and numerous candidate therapies being studied, future clinical trials are now faced with challenges related to sample size requirements, efficiency and demonstration of incremental benefit on traditional end-points in patients receiving background therapy with multiple drugs. Novel clinical trial end-points, innovative trial designs and statistical approaches and new technologies may be potential solutions to tackle the challenges fac

SUBMITTER: Weatherald J 

PROVIDER: S-EPMC11525337 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature