Rare disease trials face some of the most complex challenges in drug development. Patient populations are small and geographically dispersed. Protocols are often demanding, with intensive procedures and long travel requirements. Families are already carrying the burden of managing a...
Angi Robinson
There are as many as 7,000 distinct types of rare and genetic diseases, and an estimated 400 million people suffer from a rare disease globally. In addition, three of 10 children with a rare disease won't live to see their...
In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development,[1] addresses...