From what I have seen with orphan drugs, the smaller the population, the higher the cost of the drug. This is an observation only, I don't have time to look up the stats sorry. If the average yearly cost is $160k for an orphan drug (as someone mentioned above, haven't looked that up) then the smallest populations may be worth say $500k per year.
Ann: FDA grants Orphan for both Phelan-McDermid and Pitt Hopkins, page-24
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