Most drugs for rare diseases do have extraordinary costs associated with them by the time they get to the patient, mainly on the small target market - why the real gold ticket is PBS as most of the time unaffordable to many without it.
Biogen's Spinraza for SMA approved last year - $125K a dose and needs 6 doses in the first year and 3 in each year thereafter - so that's $725K in year 1 & $375K from year 2.
Sarepta's Eteplirsen for DMD approved last year $300K a year.
The average cost to patient of rare disease drugs is well over $100K a year though.
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