My apologies if this document has been mentioned and posted by someone before.
I came across it this morning while doing some research.
I don't recall seeing it before.
The Next Generation of Rare Disease Drug Policy Ensuring Both Innovation and Affordability
7 April 2022
https://icer.org/wp-content/uploads/2022/04/ICER-White-Paper_The-Next-Generation-of-Rare-Disease-Drug-Policy_040722.pdf
Extracts from page 19 onwards:
Noting:
"While orphan products range widely in price, a small proportion carry an extremely high price,
usually linked to very small (e.g. < 10,000) eligible patients. Research shows that orphan drugs
costing over $500,000 per year represent 5% of all orphan drugs and treat 0.08% of patients taking
orphan medications (ref 85)."
Here is the document referred to as Reference 85:
https://rarediseases.org/wp-content/uploads/2021/03/orphan-drugs-in-the-united-states-NRD-2020.pdf
GLTAHs
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