My apologies if this document has been mentioned and posted by...

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