"Developing Orphan Products: FDA and Rare Disease Day."
Happy Rare Disease Day...if you're one of 25M Americans who haz one, the government haz incentives for pharma to build you a pill.
We define a rare disease as having such a low prevalence that your family doc would only expect to see about 1 case/year.
The NIH Office of Rare Diseases quantifies a rare disease as having a prevalence of less than 200k affected patients (in the US), or 5 people for every 10k in a community.
Thank another form of Reaganomics for pumping support via the Orphan Disease Act, signed into law in 26 years ago.
Rare Disease resources:
Why rare diseases will increasingly be a focus of medical research:
1. incentives - yay ODA!
2. genomics and personalized medicine (80% are genetic in origin)
3. they hit kids (75%)
4. 1+2+3 = a PR home run for Big Pharma, who could sorely use one
Get your Rare Disease Day poster here: