HOUSE PASSES MATSUI’S RARE ACT

WASHINGTON, D.C. – Today, Congresswoman Doris Matsui (CA-07), co-chair of the Rare Disease Congressional Caucus, released the following statement after the House passed the Mikaela Naylon Give Kids A Chance Act, a package of rare disease legislation that included her Retaining Access and Restoring Exclusivity (RARE) Act. The RARE Act would ensure that promising options for rare disease patients with no approved therapies are able to come to market without being blocked by other pharmaceutical companies. 

“Today, the House delivered long-overdue hope for the millions of Americans and their families struggling with a rare disease,” said Congresswoman Matsui. “I’m proud my RARE Act is included in this bipartisan package. It will prevent pharmaceutical companies from abusing their orphan drug status to keep other innovative drugs from coming to market. As Co-Chair of the Rare Disease Caucus, I’ve met far too many families who have been told there are no options for their child’s care. This legislation brings them real hope by ensuring investment into pediatric therapies and getting promising treatments to patients faster. Now the Senate must act quickly, because for the rare disease community, every single day matters.”

Specifically, the RARE Act clarifies FDA’s longstanding interpretation of the Orphan Drug Act to ensure that the scope of the orphan drug exclusivity applies only to the same approved use or indication within such rare disease or condition, instead of the same disease or condition. This will give FDA the necessary authority to approve the same drug from different manufacturers if they aim to serve different patient populations.

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