This report reviews the development, use, and spending trends of orphan drugs in the U.S., focusing on treatments for rare diseases. It highlights how regulatory incentives have driven innovation, resulting in a growing pipeline of orphan medicines. While per-patient costs are often high, the report shows that total orphan drug spending remains a relatively small share of overall pharmaceutical expenditures. The analysis also addresses access challenges, payer concerns, and variability in coverage decisions. The report concludes that orphan drug policies have been largely effective but require ongoing refinement to balance innovation, affordability, and equitable patient access.

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