Concerns about the growth in spending on drugs that treat rare, or orphan, diseases — that it may lead to unsustainable drug expenditures — do not appear to be justified, according to a new study in Health Affairs.
For the study, researchers at IMS Health and drugmaker Celgene analyzed a database of audited biopharmaceutical sales to measure U.S. annual spending on orphan drugs from 2007 to 2013. They analyzed 356 orphan drugs approved under the 1983 Orphan Drug Act.
They found orphan drug spending totaled $15 billion in 2007 and $30 billion in 2013 —representing 4.8 percent and 8.9 percent of total pharmaceutical expenditures, respectively.
Researchers also did a future trend analysis for 2014 to 2018, which 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," they said.
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