This note once again deals with the prickly notion of how much to charge for disease altering/curing drugs which target said small populations. Recall the Orange Jesus:
America will never be a socialist country.Well... NOT!! Without explicit Socialism, albeit for for-profit capitalists, much of USofA capitalists would be up a creek without a paddle. Subsidies all over the place, and the nefarious and pernicious drug industry which has been pursuing multi-million dollar drugs which even few of the 1% could afford. Who ends up paying for these drugs? You and me, of course either explicitly through government programs or insurance premiums.
So, we get this drug from Orchard (and its now recent Japanese owner), which cures (so far supported only by short-term data, by the way) a very, very rare disease in children; if not dealt with nearly immediately, even this new drug doesn't work.
And, of course, it should be noted that Orchard abandoned a previous orphan drug 'cure'. They were not alone in taking that avenue.
So, is it worth $4.25 million? Your money or your life? Well...
Here's an estimate of the average USofA citizen's life-long income: if we take the middle value for men, adjusted, it's $1.7 million.
So, 2 and a half times your money or your life!!
And, one should also note, that Amylyx conned FDA into approving their ALS drug on lousy data, but managed to make a bundle off it in just a few years. And, by the way, 'conned' isn't just an empty epithet. Amylyx offered up lousy data and an aggresive patient community against FDA, and won. Paper tiger.
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