Case Study 1
Phase II/III rare paediatric metabolic disease, Nieman Pick
Rare diseases & paediatric
We understand the complexities of managing rare disease studies, and the determination and adaptability required from your CRO
We have a deep understanding of rare diseases many of which are paediatric studies requiring a deep desire from your CRO to overcome the complexities involved. More challenging than trials in common diseases, which is why adaptability and resilience from your CRO is crucial to getting the job done. That’s why we are focussed on patient-centric trials and novel designs to help improve patient lives, supported by a world-leading rare disease advisory board.
One team, dedicated to positive outcomes
Nathalie Ernault-Roseau, Director of Project Management
Our personal approach to project management comes with 23 years of experience in rare disease and oncology development. Discover how we can work with you.
Case Study 2
Phase I/IIa rare respiratory disease, Idiopathic Pulmonary Fibrosis (IPf)
Webinar – Orphan Drug Designation (ODD): Meeting The Unmet Need
If you missed our live Orphan Drug Designation (ODD) webinar you can view on demand now. Achieving ODD status is a significant step enabling your product to be licensed faster, cost-efficiently, and achieve market exclusivity for 10 years.
Watch nowDrug types
We have managed clinical trials with all products types including, vaccines, new chemical entities (NCEs), antisense therapy, small molecules, peptides, antibodies, genetically modified organisms (GMOs) and biologics, and gene therapy.