Case Study - Driving Regulatory Success with External Controls in Severe Infection Trial

Background

A small biopharmaceutical client faced the critical task of creating a Phase III trial design that could compare all-cause mortality (ACM) for its novel anti-infection therapy against historical controls across four distinct pathogen subgroups, while assuring FDA acceptance. The FDA confirmed it would consider a design demonstrating superiority over inadequately treated external controls.

Challenge 

Without prior experience in Bayesian design or the analysis methodologies required for externally controlled trials, the client turned to Cytel’s Strategic Consulting team to develop a study design optimized for regulatory approval. This required identifying viable design options, developing a robust analysis methodology, running trial simulations, and drafting text for the protocol synopsis. Additionally, the client needed expert contributions to the FDA Type C Meeting Briefing Book and strategic representation during the FDA meetings.

Outcome 

Download our case study to learn how Cytel’s approach led to an FDA-accepted design proposal, and trial optimization that resulted in a 33% reduction in patient enrollment and an estimated cost savings of $2.4 million.