Rare diseases & paediatric

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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 1

Case Study 1

Phase II/III rare paediatric metabolic disease, Nieman Pick

Read here

Case Study 2

Case Study 2

Phase I/IIa rare respiratory disease, Idiopathic Pulmonary Fibrosis (IPf)

Read here

Rare Diseases

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 now

Drug 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.

Let’s collaborate

If you think we could be right for you, get in touch.

Submit an RFI/RFP