title: Advancing clinical trial design in pulmonary hypertension.
abstract: In pulmonary hypertension, as in many other diseases, there is a need for a smarter approach to evaluating new treatments. The traditional randomized controlled trial has served medical science well, but constrains the development of treatments for rare diseases. A workshop was established to consider alternative clinical trial designs in pulmonary hypertension and here discusses their merits, limitations and challenges to implementation of novel approaches.
authors: Aptiv Solutions, Innovation Centre, Stevenage Bioscience Catalyst, Gunnels Wood Road, Stevenage, Hertfordshire, SG1 2FX, UK.
authors: Andy P
authors: AP
authors: Grieve
authors: Shien-Chung
authors: SC
authors: Chow
authors: John
authors: J
authors: Curram
authors: Stephen
authors: S
authors: Dawe
authors: Lutz O
authors: LO
authors: Harnisch
authors: Noreen R
authors: NR
authors: Henig
authors: Hsien Ming J
authors: HM
authors: Hung
authors: D Dunbar
authors: DD
authors: Ivy
authors: Steven M
authors: SM
authors: Kawut
authors: Mohammad H
authors: MH
authors: Rahbar
authors: Shen
authors: S
authors: Xiao
authors: Martin R
authors: MR
authors: Wilkins
keywords: Bayesian modeling; adaptive designs; clinical trial design; group sequential designs; modeling and simulation; population enrichment; pulmonary arterial hypertension
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