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TOPIC: WTF
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Fair enough on that, but I don't think that would change very much.

Again - that's terrible about your sister. I wish you all the best.

Windermere FL
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"I know. That's an orphan drug. I could explain more to you about what that means. "

I know what an orphan drug is.

I'm just saying that you don't need a marketing budget for a drug for a rare disease with ONE approved medication. Once it's diagnosed the Doctors should know what to prescribe.

Pittsburgh PA
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Firstly, I am very sorry to hear about that.

"I've read that the manufacturing cost was about 1 percent"

The manufacturing cost is insignificant compared to the development cost, which has already been spent and needs to be recouped. Without knowing anything about the development costs for this specific drug, the best I could ballpark it at was around $500 million before the first dose is sold.

".and who are they marketing to?"

I just explained that - they are marketing it to a few hundred people who have had the rather severe misfortune of developing the condition.

"It's the ONLY FDA approved treatment. "

I know. That's an orphan drug. I could explain more to you about what that means.

Windermere FL
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Thank you

Pittsburgh PA
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@Perfect: Wow, sorry.

Vero Beach FL
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My sister was diagnosed last week.

I've read that the manufacturing cost was about 1 percent............and who are they marketing to? It's the ONLY FDA approved treatment.

Pittsburgh PA
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To put it in perspective;

Occurrence is around 3 people per million. Most die within 3 years. So in a country like the US that means around 1,000 people would have it throughout their lives, and only about 100 (at MOST) would be alive at any given time. Worldwide, around 15 times that much, but at most 1/3 of those are in countries that could afford it. So you're talking around 500 "customers".

500 customers x $400k = $200 million in sales

I don't know what it cost to develop and approve, but typical these days is around $400 million. Add another $100 million for manufacturing capital expenditures, marketing etc. and this means that the manufacturer won't make their investment back for at least 2-3 years, and their exclusivity protection runs out in 2017.

i.e. even at $400k a year, they still won't make any money on the drug.

Windermere FL
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Three reasons:

Orphan drug

Monoclonal antibody

Extremely rare disease

Windermere FL
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WTF?

I had never even heard of 'aHUS', and how the fuck can a drug called Soliris cost over $400,000 a year?

Pittsburgh PA
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I haven't watched this but that may mean that Tamar Braxton is set to win.

Vero Beach FL
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TOPIC: WTF
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