Abstract

In the current economic climate there is a tightening of research income while the demand to answer important clinical questions it could be argued is the same or is even increasing. Efficient study designs enable the questions to be answered while making optimal use of finite resource.

An efficient design is defined as “statistically robust” designs that facilitate quicker and/or lower cost decisions when compared to conventional trial design. An efficient design is an umbrella term which encompasses aspects such adaptive designs and trials where routine health service data are used for the primary outcomes as well as “smarter” ways of doing trials. By being efficient in the study design resources and time can be saved in the assessment of new health technologies.

Through a series case studies the benefits of efficient study designs will be highlighted. For example: The PLEASANT trial used routinely collected data for data collection and as a result was approximately 1 million cheaper; retrospective analysis of RATPAC as a group sequential showed that if the trial had been adaptive it would have required a third of the patients and saved 250,000. An audit of public funded trials suggested that if futility were undertaken then 20% of trials would stop early and the proportion of successfully recruiting trials would increase from 45% to 64%. As well as highlighting the benefits the presentation will also discuss some of the challenges.