Rare disease clinical trials are presented with unique challenges, since treatments for rare or orphan diseases are often not available. We specialise in this area and have a deep understanding of rare disease clinical trial designs. We know the complexities involved during clinical research, with an adaptive and resilient approach to delivering positive outcomes.
Therapeutic acumen and dedication from your CRO is crucial for successful rare disease clinical research, which is why we stay committed to patient-centric and novel rare disease trial designs. We work with you to help improve patient lives with the support of a world-leading rare disease advisory board.
Due to low patient populations, recruitment for rare disease clinical research is specific and challenging. We stay committed to helping improve patient lives, developing a tailored patient recruitment strategy to maximise the reach and potential of our rare disease clinical trials.
Our team includes dedicated recruitment specialists who collaborate closely with patient groups and clients, considering geographical, practical and patient care elements to ensure patient-centric rare disease trials that deliver the best possible results.
Over 50% of rare diseases affect paediatric patients. Our rare disease clinical trial team are highly experienced with paediatric patients, and create tailored trial designs that work collaboratively within the demands placed on families. Our trial processes are designed to consider practical and geographical limitations, ensuring a fully patient-centric approach.
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.
Accelerating the needs of rare disease clinical research
Phase II/III rare paediatric metabolic disease, Nieman Pick
Phase I/IIa rare respiratory disease, Idiopathic Pulmonary Fibrosis (IPf)
If you missed our live Orphan Drug Designation (ODD) webinar you can view on demand now. Achieving ODD status is a significant step during the rare disease clinical trial process. This enables your product to be licensed faster, cost-efficiently, and achieve market exclusivity for 10 years.
Watch nowWe have managed rare disease clinical trials with all product types including, vaccines, new chemical entities (NCEs), antisense therapy, small molecules, peptides, antibodies, genetically modified organisms (GMOs) and biologics, and gene therapy. No matter the focus of your rare disease clinical trial, we get the job done.
