Cmed marks Rare Disease Day 2020

Written by Cmed

This week ends on a rare 29 February, the date selected to mark Rare Disease Day around the world.

More than 6,000 rare diseases have now been identified. Some affect only a very small number of patients, but it is estimated that 300 million people worldwide live with a rare disease. While some are the result of infections/allergies/environmental causes or are degenerative and proliferative, 80% of these diseases have genetic origins.

Cmed specializes in running clinical trials in rare diseases and we have now worked on well over 100 related clinical trials (half of them in rare oncology). This work requires specific capabilities: the most obvious is the ability to recruit patients who are often scattered around the world. Therefore, the clinical trial team not only needs to identify the optimal number of countries and sites, but also the right sites that specialize in the disease area.

Optimal protocol design is essential to narrow down the right population in ways which do not prevent enrolment and also ensure patient-friendly designs. Cmed works with experienced partners to provide patient services and where possible on a home-basis.

Cmed also has a team of medical doctors who can support in areas where there is little research data and often ‘generic’ treatments. Regulatory challenges range from understanding the approval criteria to providing substantial evidence of drug effectiveness. A lot of the studies that Cmed manages focus on gene therapy which requires expert knowledge.

Cmed has recently helped sponsors trial new treatments for Inherited metabolic diseases, Primary Biliary Cholangitis and Wilson Disease.

If you would like to talk to our team about our experience and capabilities in rare diseases, please contact in the first instance:

In North America: Jay Conze

In the rest of the world: Maria Iliescu