Matt Austin
Amgen
Amgen
Optimizing Geographic Site Distribution: Time/Cost Tradeoffs
In collaboration with Vlad Anisimov, Matt Austin outlines Amgen's approach to the fundamental question "Where should we place the study that will enroll the fastest and cost the least?". How can trial planners successfully solve this core problem that only becomes more complex as a candidate progresses through the development process?Bob Beckman
Georgetown University
Georgetown University
Design Concept for a Confirmatory Basket Trial
Bob Beckman from the Georgetown University's Medical Center presents his most recent research on balancing the theoretical and the practical considerations with basket trials. Bob begins by describing the more general design basket concept, then moves to recommendations on overcoming challenges in a more detailed manner.Peng Sun
Biogen
Biogen
Novel Design and Implementation of Interim and Final Analyses in Sham-Controlled Double-Blinded Phase 3 Trial
Biogen's own Peng Sun probes the thinking behind determining their analyses plan in developing a new Spinal Muscular Atrophy treatment. Sun and colleagues considered the statistical issues for two related confirmatory trials for infantile and later onset of the disease.Jim Bolognese
Cytel
Cytel
Adaptive Design and Trial Monitoring: a Practitioner's View
Jim Bolognese, a Senior Director with Cytel Consulting examines Potential TRUE underlying Dose-Response Curves taking into account the Tolerability in addition to Efficacy. Jim also discusses the design comparisons of tolerability to active control, focusing on opioid-induced adverse effects.Ed Whalen
Pfizer
Pfizer
Adverse Event Monitoring: Safety Statistics in Focus
Pfizer's Ed Whalen begins with an overview of safety statistics in general for randomized controlled studies, observational studies, and spontaneous report data. 2) Examine recent changes in investigational new drug (IND) safety reporting. 3) Questions illustrating the need for improved methods to evaluate important side effects.Cyrus Mehta
Cytel
Cytel
Multi-Arm Multi-Stage (MAMS) Trial Designs
Cytel President and Co-Founder Cyrus Mehta