BIO responds to Health Affairs Analysis, highlighting the importance of orphan drug incentives

Orphan drug development is one of the more fraught undertakings in biomedical research. Targeting rare diseases often requires cutting-edge science, but science is not always enough. The economics of treating small populations can make it difficult to secure the financial support needed to make it through clinical trials and onto the market.

To ensure that promising ideas aren’t abandoned in the face of those challenges, Congress has provided a number of incentives for orphan drug development, including provisions that exempt orphan drugs from government price controls under the Inflation Reduction Act.

But those incentives often come under attack. Recently, a Health Affairs article suggested that the IRA-related protections weren’t needed, given that some orphan medicines are commercially successful.

That analysis, however, is built on the flawed premise that just because a sliver of orphan drugs earn back development costs, orphan medicines, as a class, don’t face unique and daunting obstacles throughout the R&D process.

BIO believes that kind of thinking is wrongheaded, and Health Affairs this month published a rebuttal from BIO’s Vice President of Healthcare Policy & Research, Crystal Kuntz.

BIO argued that orphan drugs carry unique scientific challenges, uncertainties in development, and commercial obstacles that would discourage innovation without additional supports.

“Incentives to develop orphan drugs have been critical in encouraging research, and eroding such policies risks starving some of the best ideas in medicine of resources at the exact wrong time for patients waiting to benefit from today’s groundbreaking science,” Kuntz wrote.

BIO noted that the existence of these incentives has led to a “golden age” in orphan drug development, but that much work remains to be done. Thousands of rare diseases still lack a treatment, an indication of the need to not roll back the very policies that are making an impact.

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