Returning individual results to participants is gaining momentum as a core expression of patient-centered research. In rare disease development, where participants often contribute significant time, personal risk, and emotional investment, access to personal study data is increasingly seen as part...
Navigating rare disease drug development requires coordinated, adaptive strategies as science and regulation evolve in parallel. Small, geographically dispersed patient populations, limited natural history data, and heterogeneous disease progression make trial design and execution unusually complex. At the same time,...
Since PDUFA VII was signed into law in September 2022, the U.S. Food and Drug Administration (FDA) has introduced a series of initiatives aimed at addressing the persistent challenges of rare disease development. Collectively, these efforts form a more complete...
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...
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In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases.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...