Getting from an Interim Analysis to a Breakthrough Designation
Pablo Lapuerta — Protection + Compliance
The Moment
A client conducted a Phase 1 clinical trial where recruitment was challenging. An interim analysis was planned, but the targeted recruitment had not yet been reached.
Recruitment was so difficult that there was a temptation to do the interim analysis early, but there were concerns about how this might be perceived. The company asked for expert advise.
The intervention was an injection of placebo in one ear and active drug in the other, with the intention of preventing hearing loss associated with cancer chemotherapy. A blinded review showed that most patients had a difference between ears in audiometry (hearing thresholds) at the end of the study.
The Diagnosis
Dr. Pablo Lapuerta, grIP's domain expert, recognized the potential for a very positive clinical trial result. A specific statistical approach was recommended, with a good probability of achieving a high level of significance, even before the pre-determined interim analysis point.
The Work
The client's worry had been how an early look at the data would come across. Dr. Lapuerta's statistical plan answered that. It gave the team a scientific reason to run the analysis before hitting the recruitment target, and from there things moved fast.
With the diagnosis in hand, the interim analysis was performed early and its conduct was scientifically justified.
The results were so positive that the clinical trial was terminated early.
The database was locked, the study report was completed, two abstracts and a manuscript were prepared, and a dossier was assembled for the FDA making the case for an abbreviated clinical program.
The Outcomes
Stopping a trial early is a bet that the data will speak for itself. This time it did, and the effects reached well past the study.
- The client saved time and money with a strategic, science-driven approach to clinical development
- Abstracts were successfully presented at major scientific meetings, and the manuscript was published in the world’s leading oncology journal
- The FDA granted breakthrough designation for the program
In this case, Dr Lapuerta helped the team develop a strategic vision for the asset, execute with attention to detail, and meaningfully advance clinical development.