Clinical Trials In Rare Diseases

Developing therapies to treat rare diseases is a critical area of clinical research. However, the lack of natural history data and well-established clinical endpoints can result in complex clinical protocols that are unduly burdensome. Decentralized Clinical Trials (DCTs), provide an opportunity to transform participation in rare disease clinical trials. By moving from the traditional site-based model to DCT elements that can be brought to participants’ homes can reduce the overall study burden and improve the participant experience leading to better engagement and retention.