Take a deeper dive

Case Studies

Using Existing Pediatric Data to Guide a New Study

Background & Problem

The Sponsor’s drug had been approved in Europe for decades, but had not been developed in the US. ┬áThe Sponsor wanted to bridge the studies that were conducted in Europe with a few additional studies conducted in the US to gain approval of the drug by the FDA. Since it had been quite a while since it was approved in Europe, pharmacokinetic (PK) data were lacking.

The Sponsor enlisted our help to design a strategy that would bridge between the current study and the previous clinical experience and to help guide clinical trial design in upcoming studies.

Our Solution

Nuventra’s scientists developed a population PK model based on data from a current pediatric study and created an exposure/dose-response model to simulate which doses and which dose regimens would likely prove efficacious and remain safe in upcoming studies.

Outcome

Simulations indicated that a certain dose range in patients between 2 and 5 years old and doses up to a certain amount in non-ambulatory patients would remain safe.