[WhompingWindows]

Interesting post. Hopefully some innovators can come through and figure out a way to make it more cheaply. Even if it's expensive now, if humans exists for 100's of years, then eventually the price will come down when this inventor is toast, right? Either that or people will figure out cheaper, DIY ways to do some of this biological manipulation.

[jpalomaki]

Based on article the price is not at 1M because of high manufacturing costs, but because they want to have return on the investment made during the research and development phase.

[InTheArena]

TO be frank, it's not even that. If you read the article, they never priced it based on the what they invested in it, but rather off the the fact that they could replace something else that was expensive with it.

[tptacek]

It's not even clear that this therapy is locked up because of IPR. If you read uniQure's filings, their patents appear to be for treatments for other conditions (notably Huntingtons), and their protection for the LPLD therapy is under "orphan drug" protection.

[ineedasername]

Yeah, seems ripe for someone to pick this up and charge $100k a dose. Insurance companies would look at that as a deal compared to $300k/year for the alternative replacement therapy.

[DoctorOetker]

another issue I have is with the claim from the article that they constantly have to produce new batches because the old product expires. but with such a small group, it would seem better to first make a comprehensive list of LPLD sufferers, then make a single batch for all of them, and repeat every say 5 years as new cases are found... yeah on average a patient waits 2.5 years...

If there is a long list of rare diseases, a company could specialise in manufacturing doses for rare diseases outside of the classical "order a dose (from continuous production) as a patient is discovered". So they might make a specific rare drug once every X years, but with enough rare drugs they might fill the rest of those years with preparing for and then making and testing single batches of other rare drugs...