PZ99, thanks for your comments...
Hmm drug pricing certainly isn't a specialty of mine....the way I see it is there is a kind of amortization effect in play at least to some extent.
The less the patients, the less there is spread of costs. So in isolation treating just a few thousand patients isn't going to be cheap. A good article that covers some of the costs of ERT medicines is here:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3564370/
Is it fair that a similar drug can address OA and may ultimately cost around the $5 K mark, perhaps more, while a similar drug can cost 10 X this for a Rare indication. How seemingly unjust is it that some of these patients are paying $500,000 and may not get much benefit?
Sure there is a question of insurance and how much will be covered and who is eligible and how many can afford it which I know you have addressed in the past. You aren't wrong for taking a more conservative approach. I for one cannot say how it will work out for us but at the end of the day I see how the drug works for most (not all), I see the good side of it and the relative lack of side effects and certainly AE's at this early point and that gives me more confidence.
Understood you need two parts of the puzzle to come together, the efficacy and the commercial aspects. There are many that have had a great product but due to mismanagement, well it's all evaporated quickly. Not saying that will happen to us, I am saying that we need to be aware of that side too.
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