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The Inflation Reduction Act (IRA) exempts orphan drugs for rare diseases from price controls—but only if the drugs are approved for a single indication. The ORPHAN Cures Act—currently under consideration in the House and Senate—would fix the law, and industry and American voters overwhelmingly support it.
Orphan drugs target rare diseases. There are around 7,000 such rare diseases, but each one typically affects fewer than 200,000 people in the United States, we’ve reported.
Of the 280+ orphan drugs approved since 2003, nearly a quarter were approved for additional indications. However, while the IRA would exempt drugs developed for a single rare disease indication from price controls, drugs that are approved for two or more indications could still be subjected to the government’s price-setting structure.
“By definition, orphan drugs benefit small patient populations, making investment in this space incredibly risky. But there is tremendous need for these treatments. Fewer than 5 percent of rare diseases have [a Food and Drug Administration] approved treatment,” explained Biotechnology Innovation Organization (BIO) CEO Rachel King in September.
How the ORPHAN Cures Act could incentivize R&D
As we’ve reported, the Optimizing Research Progress Hope and New Cures (ORPHAN Cures) Act would change the IRA’s incentive structure to encourage follow-on development, meaning development for more than one indication.
The ORPHAN Cures Act was introduced in the House in September, and in the Senate in October. The legislation is supported by BIO, as well as the Council of State Bioscience Associations (CSBA), the coalition of state-based BIO affiliate organizations.
“If enacted, the ORPHAN Cures Act would support existing incentives and boost research into new treatments by incentivizing research in follow on indications,” said a recent CSBA letter to congressional leaders.
American voters support orphan drug R&D
American voters support the ORPHAN Cures Act and want to incentivize rare disease drug development, found a Morning Consult poll commissioned by BIO.
The poll, which sampled 1,990 registered U.S. voters between Nov. 23- 25, found 94% of voters believe rare disease R&D is important. In addition, 4 in 5 believe incentives for rare disease treatments should be a critical policy priority.
A majority of surveyed voters also support legislation, like ORPHAN Cures, to encourage rare disease development and experimentation, especially treatments for multiple indications. In fact, two-thirds of those polled want their lawmakers to vote for the bill.
“Support for government action on rare disease research is bipartisan,” says the poll. “The impact of incumbent representatives voicing support for the ORPHAN Cures Act is net-positive for Republican, independent, and Democrat voters alike.”
“Right now, 95% of rare diseases have no FDA-approved treatment. As this poll shows, Americans want lawmakers to incentivize the scientific pursuit of these much-needed treatments,” said BIO CEO Rachel King.
