Futility Analyzes in Confirmatory Clinical Trials – Methods and Procedures (ADDED FEE) — Professional Development Continuing Education Course
ASA, Biopharmaceutical Section
Futility analyses (FA) are increasingly utilized in clinical trials. FA generally involves interim evaluation of a trial’s primary hypothesis to determine if trial success or clinically meaningful effect seems unlikely. FA can improve resource efficiency by halting trials with ineffective intervention. They also offer ethical advantages by exposing fewer trial participants to ineffective and possibly toxic interventions, and public health advantages in that trial results may be conveyed to the medical community in a more timely fashion.
FA should be carefully planned during trial design phase, as there are important statistical and operational consequences. Concerns include the control of statistical error rates and the potential for operational bias resulting from interim evaluations. Challenging questions arise during trial design regarding how FA should be conducted, when futility should be assessed, role of Data Monitoring Committees and thresholds at which futility may be established. Non-constancy of effect size and other limitations of accruing interim data can raise further challenges.
In this course, we will describe current practices and recent advancement in methodological approaches of FA with case studies. We describe what FA are, why they are conducted, where and when they should be considered, and how they should be methodologically and operationally performed.
Instructor(s): Satrajit Roychoudhury, Pfizer Inc ; Paul Gallo, Novartis Pharmaceutical