fda orphan drug web resource

The FDA’s New Orphan Drug Web Resource – continued commitment to rare disease research

In Advocacy, Regulation & Government by Caroline Hornby

A new web-based rare disease resource for patients and industry was recently launched by the U.S. FDA’s Office of Orphan Products Development.

View the new website here.

The website contains information on:

  • Designating an orphan product: How to apply for orphan drug designation
  • Orphan products grants programme
  • Designating Humanitarian Use Device (HUD)
  • Paediatric device consortia grant programme
  • Rare pediatric disease priority voucher programme
  • OOPD news, events and archives

This new website arguably demonstrates the FDA’s commitment to rare disease research and orphan drugs.

As reported by Delaware Online,  the FDA’s director for the Office of Orphan Products Development, Gayatri Rao, said “while many factors may be contributing to the growth of orphan drug development, patients are continuing to drive the push for innovation and treatments. Last year, the FDA funded 15 new orphan products grants for about $14 million, all supporting clinical research in rare diseases.” It also approved 33 drugs for rare diseases, compared to just ten years prior to the Orphan Drug Act when less than 10 rare disease treatments had been developed.