Rare disease trials face unique challenges. For sponsors it may represent all – or a significant part – of their portfolio. For patients, clinical trials may be their only opportunity to access a potential treatment.
Until now, there has been no research to understand the participation support needs of rare disease patients and caregivers.
The results of focus groups we conducted with ICON (formerly PRA) provide insights to the gap between the clinical trials experiences and perceptions of patients and the participation support services provided to them.