Advancing clinical trial design in pulmonary hypertension

作者：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^*

来源：Pulmonary Circulation, 2013, 3(1): 217-225.

DOI：10.4103/2045-8932.109933

• pulmonary arterial hypertension
• clinical trial design
• adaptive designs
• modeling and simulation
• Bayesian modeling
• group sequential designs
• population enrichment

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.

• 出版日期2013-1