Statistical Considerations for Studies with a Performance Goal

• Activity Number: 624 - Trial Design and Analysis Issues in Medical Devices
• Type: Contributed
• Date/Time: Thursday, August 3, 2017 : 8:30 AM to 10:20 AM
• Sponsor: Section on Medical Devices and Diagnostics
• Author(s): Xu Yan* and Heng Li and Yun-Ling Xu
• Companies: U.S. Food and Drug Administration and U. S. FDA and U.S. FDA
• Keywords: performance goal ; Medical Device ; Single arm study

Abstract

In the safety and effectiveness evaluation of medical devices, randomized controlled trials (RCTs) provide the highest level of evidence. However, it is not always feasible to conduct an RCT due to practical or ethical reasons. As such, a single arm study in which comparison against a pre-specified performance goal is made plays a substantial role in pre-market device evaluation. While this type of study design has potential benefits, such as savings in cost or time, statistical challenges arise regarding the validity of study design and the interpretability of study results. In this presentation, we will discuss these challenges, focusing on issues with weighted performance goal.