Case Studies

Cost Savings as a Result of Sample Size Reduction

• Early termination of a phase 2b and extension trials using Bayesian posterior predictive probability - a 50% cost saving
• Sample size re-estimation using Bayesian posterior predictive probability - a 30% reduction in trial length

Rescue Mission for an NDA Submission

• NDA re-prepared with amended phase 3 CSRs, new ISS, new statistics data package compliant to the FDA eSubmission requirement - all completed in 2½ months

Consideration in Study Design

• Mid-course modification of a group sequential trial from the O'Brien-Flembing "spending function" to an ad hoc Lan-DeMets spending function
• Non-inferiority study design for a cardiovascular drug with MACE endpoint - a sample size challenge