FDA Moves To Rein In Drugmakers' Abuse Of Orphan Drug Law

[Cllcanada]

All CLL drugs are developed under this act...

Over three decades ago, the United States Congress passed the Orphan Drug Act (ODA) in 1983. The act focused on encouraging drug development for rare diseases, which the agency defined as “affecting fewer than 200,000 people.” When new FDA commissioner, Scott Gottlieb, M.D., joined the FDA in 2016 he promised to revamp the ODA and close abuse-prone loopholes.

npr.org/sections/health-sho...

Wikipedia

en.m.wikipedia.org/wiki/Orp...

PLOS

ncbi.nlm.nih.gov/pmc/articl...

~chris

[Jm954 Administrator]

That doesn't sound in the spirit of the orphan drug/disease designation, very naughty.

The NHS uses a definition of a prevalence of 0.18/10 000 or less which sounds more generous than the FDA. NHS commissioning took the enlightened view that they would pay premium prices for drugs for these conditions but NICE still does the scrutiny.

One of them is PNH (paroxysmal nocturnal hemoglobinuria) which is a clonal disorder that causes haemolysis, pancytopenia, renal failure and thrombosis. Cost of the monoclonal antibody Eculizumab was approx £340,000 per patient for life!