Rare Disease Day 2011

Today, February 28, is Rare Disease Day.  At first blush the event sounds a little obscure, but not so much when you learn that 30 million people in the United States suffer from at least one rare disease.  Unfortunately, of the rare diseases that are known, 95% do not have an FDA approved drug treatment.

That might start to change in the coming years.

Due to changing regulatory, reimbursement, and competitive dynamics, many pharma companies are entering into the rare disease space which was formerly the domain of small to mid-cap biotechs such as BioMarin, Genzyme, and Shire.  Most notably, Pfizer announced the formation of a rare disease group within their organization.  

I applaud the efforts of those involved with Rare Disease Day and encourage people to check out the accompanying website.