In a world with more than 6,000 rare diseases and very limited available treatment options, developing medicines to treat rare disorders is very exciting and satisfying. However, conducting studies in rare diseases is not straightforward as it brings distinctive challenges in terms of study design, regulatory review and approval processes, identifying and setting up the right sites, recruitment of small number of patients, retention and logistical considerations. As a result, rare disease trials require a completely different and innovative thought process and creative solutions compared to running standard clinical trials.
One of the most important elements associated with the success of any clinical trial is recruitment and then subsequent retention of patients for the study duration to ensure collection of complete data sets. This becomes especially vital when running studies in rare diseases. It is a known fact that rare disease patients are scattered across countries and for many rare diseases the number of patients is extremely limited. In such cases a clinical trial may involve setting up numerous sites in different countries across the globe with each site contributing one to two patients in order to meet the required number of patients.
- Study Protocol and Visit Schedule
- Travel for Patient and Caregiver
- Home Support
Meet Orphan Reach at this year’s World Orphan Drug Congress USA 2015.