Advancing Clinical Trial Design in Pulmonary Hypertension. (1st January 2013)

• Record Type: Journal Article
• Authors: Grieve, Andy P.; Chow, Shien‐Chung; Curram, John; Dawe, Stephen; Harnisch, Lutz O.; Henig, Noreen R.; Hung, Hsien Ming J.; Ivy, D. Dunbar; Kawut, Steven M.; Rahbar, Mohammad H.; Xiao, Shen; Wilkins, Martin R.
• Is Part Of: Pulmonary circulation. Volume 3:Number 1(2013)
• Page Start: 217
• Page End: 225
• Publication Date: 2013-01-01
• Subjects: pulmonary arterial hypertension -- clinical trial design -- adaptive designs -- modeling and simulation -- Bayesian modeling -- group sequential designs -- population enrichment; Pulmonary circulation -- Periodicals; Pulmonary circulation; Electronic journals -- Sciences; Periodicals; 616.24005
• DOI: 10.4103/2045-8932.109933
• Languages: English
• ISSNs: 2045-8932

Abstract

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.