Signant’s Solutions Support Endpoint Reliability in Successful Pediatric Rare Disease Trial

Date Published:
Case Study

A pharmaceutical sponsor conducting an important trial investigating a novel treatment for a pediatric rare disease needed to ensure the accuracy and reliability of its endpoint data.

Recognizing that their current vendor lacked the expertise, the sponsor engaged Signant to provide endpoint reliability solutions and services.

As global leaders in CNS, rare disease, and pediatric trials, Signant was prepared with proven solutions, scientific expertise, and experience rescuing studies to take the lead.

By redesigning the sponsor’s rater training program and applying our Blinded Data Analytics solution, we ensured that data derived from complex assessments were accurate and ready for regulatory review, ultimately leading to FDA approval of the first novel treatment for the rare pediatric disease.

Read the case study for details, and contact our team if your trials are in need of rescue.

Download Case Study