"Orphan drug" status abuse by manufacturers

[Lexington2012]

I keep reading articles about how drug companies are able to "abuse" the orphan drug program, but they never seem to mention specifics.

I know that one is example is Crestor, but that doesn't seem like it was one that worked in their favor. It was just an unsuccessful attempt. Does anyone know of any actual specifics of the orphan drug program being abused, or is it all just a bunch of media BS?

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[lord999]

Yes, the short story is that orphan drug can be requested, but FDA has to approve it. There's abuse of the application process, but FDA is pretty good about not letting O approvals get out of hand (Epoetin alfa I think is the example that made a lot of the FDA internal regulators pissed off when Amgen screwed them over which is why the designation is a lot harder now). There's kind of a special section for PhRMA for the legitimate ones.

By the way, the law changed to make P and O are mutually exclusive requests. So, getting a P might actually be a better decision tactically even for an Orphan drug if it knowingly has broader implications once the door is opened (Sidenafil was like that where it was originally going to be submitted as a type O for pulmonary hypertension in neonates, but it's broader application changed during testing and Pfizer withdrew the O request for the broader market for erectile dysfunction).

 

[catalyzt]

Wow. How was Astra-Zeneca going to try to get rosuvastatin designated as an orphan drug?

 

[lord999]

Genetically linked familiaral hypercholesterolemia refractive to standard therapy. That was the theory when they applied. It's actually not for an approval reason (it's obviously already approved for a wider diagnosis), but for tax breaks that the US gives orphan manufacturing. The longer story is something for Pharmaceutical Regulation class.

Sidenafil actually does have an orphan designation now for the neonatal pulmonary hypertension, and when it is sold as an IV, parents sing hosannas as what was usually a fatal condition in a premature delivery is now easily treatable, and Pfizer is just as happy at the tax offset (something like 2000% (two thousand percent) of the cost of manufacturing) for doing the "right thing". Such is the tax code in this era.

 

[Lexington2012]

By the way, the law changed to make P and O are mutually exclusive requests.

O=Orphan
P=????

 

[RxPreceptor]

P=Priority