Showing posts with label rare disease. Show all posts
Showing posts with label rare disease. Show all posts

Thursday, July 8, 2010

A striking point...

On June 29, the following appeared in an article titled FDA Weighs Rare Disease Drug Regulation But Agency Nixes New Division in the publication, FDA Week.

“Experts suggest that there are roughly 200 FDA-approved treatments for the nearly 7,000 rare diseases that exist globally. So while there have been many successes, the unmet needs are huge,"
I read these words and read them again. 

Why were they so striking to me?  Do the math and consider this...

With 8 treatments for PH, 4% of the approved treatments for the 7,000 rare diseases are for PH.

While PH is a rare disease, our patients' and families' focus on putting a spotlight on this disease has brought it out of the dark corners and into the attention of our dedicated researchers and clinicians and the pharmaceutical companies considering new treatments to pursue.

We are not yet where we need to be...at this point there is not a cure.  However, these numbers  are the result of hard work toweards a clear target.  They.are one indicator that we are moving forward rapidly thanks to the dedication of our community of hope.