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Re: jbog post# 186248

Saturday, 01/17/2015 5:52:37 PM

Saturday, January 17, 2015 5:52:37 PM

Post# of 252754
Orphan Drugs:

I am in the camp that looks at the other side of the picture. What would you consider a fair price for Orphan drugs, in particular ones that are either actually extending life and/or greatly improving QOL?

I am of the belief that we wouldn't be seeing so much investment in Orphan drug development if not for the extremely high prices. If this changes I think you take a big motivation out of development and while there may still be some it would greatly diminish the effort here.

I think it will take some time to change too as I think society is of the belief that for many (most?) of these indications "we" owe these people a chance to improve their life. I've followed some of the press x-US where healthcare is rationed and when governments do not pay for some patients the public comes out against government doing so. There are several of these cases that I recall. Two recent are Myozyme for Pompe and Esbriet for IPF (and I think Esbriet is not anywhere in the league of Myozyme in terms of QOL/efficacy benefit).
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