Taking The Complexity Out Of Rare Disease Trials For Small To Mid-Size Biotech Sponsors

Rare diseases are by their very nature, uncommon. Approximately 90% of rare diseases still have no effective treatments. Disease-specific barriers to therapeutic success include small populations, disease sub-types, and diagnosis challenges. These barriers can result in an incomplete understanding of disease pathophysiology even when there are approved treatments available. Because of this, critical clinical trials in rare diseases can be difficult to run due to a lack of patients, complicated study designs and challenging logistics; all of the above imply involvement of numerous countries, many sites and lengthy timelines to completion.

 These challenges in conducting Rare Disease clinical trials are misconceptions when you have the required expertise; studies in rare diseases are collaborative, innovative and perhaps some of the most rewarding for those of us having the chance to contribute to this research. It’s clear that what is essential to success is ensuring there is the right clinical team in place. Cmed has an established rare and orphan disease portfolio and has successfully delivered 128 studies over the last 18 years; 11 of these products now have approval which is critically important for the over 300 million people living with a rare disease.