Ultra-Rare Disease And Clinical Research– Different Approaches For Unique Studies

In a previous blog, we discussed ultra-rare diseases and the unique research challenges they pose to the development of new therapies. The nature of rare and ultra-rare disease means that drug development pathways and the types of studies commonly conducted can differ from the pathways and approaches used for more common diseases.

In this piece, we will discuss some of the approaches commonly taken to bring new ultra-rare disease therapies to market, and how decentralized clinical trial (DCT) elements help.