We specialise in orphan, rare and oncology diseases. Rare diseases affect 1 in 10 people, meaning more than 350 million people worldwide*. These people are often overlooked as the exceptionally low incidence of these diseases creates significant challenges in the design and execution of clinical trials for new therapeutics.

We understand those challenges and are proud that our dedicated global team of world-class experts and specialists have probably more orphan product and cell therapy experience than any other clinical research organisation.

Our team passionately believes that those affected by orphan, rare and oncology diseases should not feel marginalised in society by their conditions. That’s why we’re dedicated to bringing therapies to market that will allow those affected to live as normal a life as possible.

*Source: https://www.rareundiagnosed.org/rare-disease-facts/

Orphan, Rare and Oncology Drug Development

We have exceptional experience in all aspects of the development of orphan, rare and oncology products, including:

  • Orphan drug designations (ODD): we’ve written and submitted more than 200 successful ODD applications to the FDA, EMA, Australian and Japanese authorities

  • Liaison with rare disease experts and patient organisations

  • Full-service support and management of global clinical trial programmes

  • Advising on pre-approval requirements for compassionate/named patient use sales

  • Planning clinical development strategy

  • Product marketing approval and beyond

Some of the 200+ orphan disease areas we’ve worked in include:

  • Rare oncology indications, such as mesothelioma and multiple lymphomas

  • Urea cycle disorders

  • Cystinosis

  • Cystic fibrosis

  • Duchenne’s muscular dystrophy

  • Porphyria

  • Alpha-1 antitrypsin deficiency

  • Pompe disease

  • Stargardt’s disease

  • Epidermolysis bullosa

  • Systemic sclerosis

Get In Touch

Our team is ready and willing to support your clinical research needs for orphan, rare and oncology diseases.

To discuss your specific project requirements, contact us today.