Orphan drugs
- Drugs with U.S. orphan-drug designation
- Target rare diseases/conditions
- Designation is regulatory—not efficacy ranking
- Price and evidence base may differ from common drugs
Class at a glance
- Drugs with U.S. orphan-drug designation
Practical pearls
- Price and evidence base may differ from common drugs
Other practical pearls
- Target rare diseases/conditions
Clinical pearls
- Designation is regulatory—not efficacy ranking
Self-check
- Does orphan designation rank efficacy?
No; it is a regulatory designation. - What conditions do orphan drugs target?
Rare diseases/conditions.
Drugs in this class (15)
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