FDA approves treatment for disease affecting fewer than 150 worldwide

http://www.raredr.com/news/fda-approves-mepsevii

"'This approval underscores the agency’s commitment to making treatments available to patients with rare diseases,' said Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research (CDER)"

It's great to see the FDA focussing on exceedingly rare disorders, but where is the commitment from the FDA to approve treatments for common diseases as well?

Fewer than 150 patients worldwide (and perhaps fewer than 7 patients in the United States) might benefit from the FDA approval of Mepsevii.

What about the 20,000,000 worldwide ME/CFS sufferers still with no approved treatment? Where is the FDA commitment to them (us)?

Priorities seem out of balance at the FDA.

 

Amen

 

Wow, I am guessing their advocacy group and scientific advisory board must be stellar in order to gain funding and get Pharma onboard.

 

The FDA did not invent this drug, they just allowed it for human use in the US

 

Yes, exactly! Ultragenyx Pharmaceutical Inc invented the new drug, and the FDA approved its use for US consumption.

http://www.ultragenyx.com/

 

"Sly syndrome"

 

The FDA also affirmed they have a "commitment to making treatments available to patients with rare diseases". (ETA: Which is a noble goal, as many rare disease have no treatment yet.)

What about an FDA commitment for non-rare diseases that don't yet have a treatment?

 

What coincidence

You'll get no argument from me, but they are going to need a drug to approve and the documentation they require. The FDA is not an evil beast, in fact they respond quite well to industry malfeasance in getting drugs approved as long as its subtle which is not actually a good thing...