In fact, ARIAD is a "poster child" for the Orphan Drug Act
-Small patient population
-Addresses unmet needs for life-threatening disease(es)
-Most of the revenue goes towards R&D and Patient Care
-Company is not yet profitable
-ARIAD has superior plan in place to ensure that All qualified patients have access to the drug, even at a loss to ARIAD (so much for the "greedy" appellation...)
-When Iclusig was pulled, all patients in the US received the drug under individual INDs, at NO COST to the patients (another example of "greed"?)
-Now close to approval for another potentially life-saving drug - brigatinib
Wow! Wouldn't it be great to present this to the Congress and to the press - amazing free publicity for ARIAD and it's life-saving ethical drugs!
The orphan drug act was put in place to allow companies exactly like ARIAD to recoup their investments and make a profit, as an incentive to develop drugs for orphan diseases.
I hope that the patient advocacy groups and doctors are already communicating this to their congressional representatives and to Bernie Sanders, et al. If little ARIAD is on the cross-hairs, then no orphan drug company is safe...
Jack Klugman (Who played Quincy, the Pathologist-detective) must be turning in his grave, as he was a major advocate for getting the Orphan Drug Act submitted and approved. There even was an episode of Quincy in the '70s that specifically covered the challenges in getting drugs developed for orphan diseases.