Randomised Controlled Trial

Client Context

A pharmaceutical company was conducting a Phase III randomised controlled trial (RCT) comparing a new antihypertensive medication against an active comparator across 12 sites in the UK and Ireland. The sponsor needed an independent statistical partner to design the analysis plan and deliver regulatory-ready results.

The Challenge

The trial had a complex adaptive design with two interim analyses planned for efficacy and futility. Sample size re-estimation was required at the first interim look, and the primary endpoint (change in systolic blood pressure at 12 weeks) needed to account for baseline covariates and site effects. The sponsor also required results in a format suitable for both the MHRA and EMA submissions.

Our Approach

We developed a detailed statistical analysis plan (SAP) before database lock, specifying the primary analysis (ANCOVA adjusting for baseline and site), multiplicity adjustments for secondary endpoints, and pre-specified subgroup analyses. We implemented the group-sequential design using O'Brien-Fleming boundaries and conducted the interim analyses through an independent data safety monitoring board. All analyses were performed in R with reproducible scripts and full audit trails.

Results

The first interim analysis met the efficacy boundary, and the trial was stopped early after 340 of the planned 500 participants. The treatment group showed a statistically significant reduction of 8.3 mmHg (95% CI: 5.1 to 11.5) in systolic blood pressure compared to the comparator. The early stop saved the sponsor an estimated 18 months and a significant proportion of the trial budget. Results were published in a peer-reviewed journal and the regulatory dossier was accepted without statistical queries.

Client Testimonial

"AnalyticsScholar delivered clarity where we had complexity. Their interim analysis was so thorough that the DSMB had no follow-up questions, and the early efficacy finding saved us substantial time and resources."

— Chief Medical Officer, PharmaSight Ltd

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