Cromos Pharma on rare disease drug development

Cromos Pharma on rare disease drug development

An estimated 475 million people are affected by rare disease worldwide (WEFORUM), and only 5% of people with a rare disease have a treatment. This represents a significant unmet medical need. Rare disease clinical trials involve challenges above and beyond those encountered for large scale clinical studies for more common diseases.

Operational and scientific challenges include:

  • Limited pool of eligible patients;
  • Wide geographical spread of study subjects and Investigators;
  • Large heterogeneity in patient populations with different phenotypes and various disease pathophysiology;
  • Lack of preceding clinical trials to establish a baseline standard for study execution;
  • Large heterogeneity in treatment effects;
  • Uncertainty in regulatory practice and various regulatory requirements in each country of operations.

Cromos Pharma overcoming challenges associated with orphan drug development

At Cromos Pharma we have extensive experience in managing all aspects of clinical trials for rare diseases. We take an innovative and flexible approach to ensure the success of our sponsors’ rare disease clinical projects. We do this by:

  • Understanding patient pathways to allow us to effectively identify and recruit patients;
  • Leveraging our extensive site and investigator network to identify the optimal sites and staff;
  • Providing ongoing monitoring and support to sites;
  • Employing effective strategies to retain patients throughout studies.

In the last 16 years, Cromos Pharma has contributed to the analysis, design, management and/or conduct of 20+ studies in rare diseases, covering Phases II to IV. Partnering with us means working with an international CRO with a strong track record in innovative trial design, exceptional multi-country patient recruitment and proven regulatory expertise.

Read our Rare Disease Drug Development Factsheet here

Contact us today at to arrange an introductory meeting and find out how Cromos Pharma can support your next clinical project.



To arrange an introductory meeting and find out how our experience can benefit your next clinical project.