April 7, 2014
A recent press release from the FDA’s Office of Orphan Products Development (OOPD), in collaboration with the Center for Drug Evaluation and Research (CDER), announced the launch of web-based educational resources for patients and industry on FDA-related rare disease topics, the first of which was revealed on Rare Disease Day, 28 February, 2014. The resources covered on the website elucidate mechanisms to interact with the agency and access therapies that are currently being studied. They have also released an FDA Voice Blog focusing on initiatives related to pediatric rare diseases. This is part of FDA’s ongoing efforts to bring effective treatments for rare disease patients, which have been commendable to date. FDA has reiterated the importance of a listening to the comments and concerns of patients and interacting with them in order to address their needs and better serve them.