Orphan drug expenditures in the United States: a historical and prospective analysis, 2007–18

Health Affairs

7 September 2016 - Divino et al. used the IMS Health MIDAS database of audited biopharmaceutical sales to measure US annual spending on orphan drugs in the period 2007–13, and to estimate spending on the drugs for the period 2014–18.

They identified 356 brand-name orphan drugs that were approved by the FDA in the period 1983–2013.

In 2014 dollars, expenditures on orphan drugs totaled $15 billion in 2007 and $30 billion in 2013—representing 4.8% and 8.9% of total pharmaceutical expenditures, respectively. A future trend analysis for the period 2014–18 suggests a slowing in the growth of orphan drug expenditures.

The overall impact of orphan drugs on payers’ drug budgets is relatively small, and spending on orphan drugs as a percentage of total pharmaceutical expenditures has remained fairly stable. Concerns that growth in orphan drug expenditures may lead to unsustainable drug expenditures do not appear to be justified.

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Michael Wonder

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Michael Wonder