Rare disease clinical trials

We understand the complexities of managing rare disease studies, and the determination and adaptability required from your CRO

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.

A collaborative research approach

As rare disease specialists, we are proactive in understanding the specific needs of your orphan drug development program. Our committed rare disease Project Managers and specialists work as an extension of your team, enabling efficient communication and strong collaboration. 

By working together, we will devise an optimal rare disease clinical trial design, focusing on study delivery that meets your specific objectives.

Full rare disease clinical trial delivery

We have over 25 years’ experience in driving complex rare disease studies, with an in-depth understanding of the science, potential regulatory limitations and operational challenges involved in orphan disease research. 

From conception to completion, we design, manage and assess your rare disease clinical trial programme across early to final phases; devised for the specific challenges of rare disease clinical research, and always with an appropriate mitigation plan.

Patient recruitment strategy

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.

Paediatric studies

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.

Handling rare disease clinical trials with expertise


We have over 25 years experience in rare and orphan diseases and can handle complex clinical trials. We conduct phase 1-3 trials, including tailored patient recruitment strategy.

Whitepaper - Practical Steps to Achieve Orphan Drug Designation Status

Whitepaper - Practical Steps to Achieve Orphan Drug Designation Status

Accelerating the needs of rare disease clinical research

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Case Study 1

Case Study 1

Phase II/III rare paediatric metabolic disease, Nieman Pick

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Case Study 2

Case Study 2

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

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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 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 now

Orphan drug types

We 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.

Let’s collaborate

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

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