Just finished up watching the FDA hearing webcast from earlier this week.
Loving Sen. Sam Brownback ….
If you have a rare disease, “God bless ya” because we “got no treatment for you.” The billion dollar per drug model doesn’t work for 7,000 rare diseases. We need a new different model – adjust the system so that it costs less and still provides quality drugs. Time to stop walking by the sick people. We can do better.
and Sen. Mark Pryor.
Not so sure how much we love about 50 minutes into the webcast. Ouch. Diane Dorman of NORD referring to Emil Kakkis of Kakkis EveryLife Foundation‘s specialized review division as an “additional silo.”