title: A framework: make it useful to guide and improve practice of clinical trial design in smaller populations.
abstract: The increased attention to design and analysis of randomised clinical trials in small populations has triggered thinking regarding the most appropriate design methods for a particular clinical research question. Decision schemes and algorithms have been proposed, with varying starting points and foci. Parmar et al. (BMC Medicine 14:183, 2016) proposed a framework designed to assist the clinical trial team in design choices during protocol preparation. Herein, further stimulus is given regarding the extent to which a framework may help change practice for the better, the careful considerations for changing the usual error levels applied and the room for innovation in clinical trial design.Please see related article: http://bmcmedicine.biomedcentral.com/articles/10.1186/s12916-016-0722-3 .
authors.affiliation: Julius Centre for Health Sciences and Primary Care, Biostatistics and Research Support, University Medical Center Utrecht, Utrecht, The Netherlands. k.c.b.roes@umcutrecht.nl.
authors.forename: Kit C B
authors.initials: KC
authors.lastname: Roes
mesh_terms: D000465:Algorithms; D002986:Clinical Trials as Topic / Q000379:methods*; D006801:Humans; D012107:Research Design
keywords: Clinical trial design; Protocol development; Randomised clinical trials; Rare diseases