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.
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...
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...