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Orphan Drug Spending: Could Sequence Of Indications Offer Pricing Policy Solution?

 
• By Sarah Karlin-Smith

Focusing on orphan-first drugs may be best route to address pricing concerns, Health Affairs study suggests after finding that just one-fifth of spending on 15 top-selling partial orphan drugs was for the drugs’ rare disease indications.

mostly wooden stick figures with one blue stick figure
Partial-orphan drug spending and policy solutions are outlined in new Health Affairs study • Source: Shutterstock

Policy makers worried about the costs of drug for rare diseases may want to hone in on “partial orphans” and focus their solutions on drugs that were first approved for a rare disease indication and then received indications for more common diseases, a new study argues.

The high cost of rare disease treatments has long been a concern for policy makers with partial-orphan drugs – those approved for both rare and common diseases – in particular...

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