Orphan Drug Status

The Orphan Drug Act

The Orphan Drug Act was introduced to encourage companies to invest in cures for rare diseases that otherwise may not be commercially attractive.

Promising drug candidates for rare diseases, such as Motoneurone disease, are eligible to be granted Orphan Drug status by the FDA.

Orphan Drug status enables the fast-tracking of promising drugs for diseases that have fewer than 200,000 sufferers and for which there are no effective treatments available.

With Orphan Drug Status it is possible to get FDA approval with only 2 phases of clinical trials.

The clinical phases are condensed with Phase I & Phase II combined into a Phase I/II trial and parts of Phase II and Phase III are combined into a Phase II/III trial. This significantly reduces the time and investment needed to get a drug to market.

Benefits of Orphan Drug Status include:

1. Seven year US and 10-12 year European market exclusivity for the developer of an orphan drug.
2. Grants available from the FDA.
3. NDA fee waivers.
4. Tax credits for development costs.
5. FDA assistance in the design and planning of clinical trials.