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Avik Roy has an absolutely blood-boiling section of this report devoted to ways companies are gaming the Orphan Drug Act

https://docs.house.gov/meetings/GO/GO00/20190129/108817/HHRG...



Not really. While he makes it sound bad, what is happening is that in exchange for actually proving that drugs we "know work" actually work companies get a monopoly for a short time. I'm glad some of the treatments doctors have been using for years now have science behind them so we know more about how well they work.




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