Posted by Michael Wonder on 19 Jan 2020
FDA harnesses technology and collaboration to support rare disease product development
17 January 2020 - Announcing an orphan drug technology modernisation effort and the 2020 FDA Rare Disease Day meeting.
Patients and their families are the focus of our work at the FDA. We ensure that treatments for diseases are safe and effective. Patients and their families are experts in their diseases and help inform many aspects of medical product development.
This is especially notable in rare diseases, where product development can be challenging given the small number of patients affected with a given disease and the potential for limited understanding of the pathophysiology of some rare diseases.
