Keywords: clinical validation, follow on diagnostic, bridging, clinical efficacy, concordance, diagnostic agreement
Room: McKinley
In the framework of follow-on companion diagnostic devices one of the key performance criteria for the diagnostic test to be shown is the clinical efficacy. In other words, the link between the test result from the diagnostic test and the clinical outcome after treatment with a specific drug has to be clearly demonstrated. In the ideal world, a clinical study would have to be conducted in order to prove a significant difference between treated and untreated patients in either the mutant-positive or the mutant-negative patients. However, in real life given the cost and the effort related to such clinical trial other strategies have been developed as clearly described in Meijuan Li 2015 and 2016. Both the strategy where patient samples of the original clinical trial were used and the strategy where an external concordance study is used trigger additional risks, which should be taken into account while planning for submission of such follow-on companion diagnostic device. The topic of the round table consists of 2 aspects: - Understanding the differences between both approaches with advantages and disadvantages associated and some discussion on yet other alternatives. - Consideration of the risks associated with such strategies and how to better cope with possible mitigating actions. References: • Meijuan Li (2015) Statistical Consideration and challenges in bridging study of personalized medicine, Journal of Biopharmaceutical Statistics, 25:3, 397-407 • Meijuan Li (2016) Statistical methods for clinical validation of follow-on companion diagnostic devices via an external concordance study, Statistics in Biopharmaceutical Research, 8:3, 355-363