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WASHINGTON, DC—Congressman Paul D. Tonko announced that his Helping Experts Accelerate Rare Treatments (HEART) Act passed out of the House last night under H.R. 7667, the Food and Drug Amendments Act. Tonko’s legislation strengthens the U.S. Food and Drug Administration (FDA) review process for drugs that treat rare and ultra-rare diseases by directly involving scientists and doctors with expertise throughout the review process. 

“Accelerating and strengthening the process of reviewing treatments for rare diseases would offer life changing access to the millions of Americans and their loved ones living with rare and ultra-rare diseases,” Congressman Tonko said. “For years I’ve pushed for this legislation to ensure expert and patient perspectives are at the forefront of the FDA’s review process. These common-sense measures will provide invaluable insight and hope of new, effective treatments. I urge the Senate to pass this bipartisan legislation without delay.”

Tonko was inspired to introduce this legislation through his work with Melissa Goetz, constituent and Co-President of The Familial Chylomicronemia Syndrome (FCS) Foundation, whose daughter is living with FCS.

The HEART Act:

• Requires a study on sufficiency and use of FDA mechanisms to incorporate patient/clinician perspective in FDA processes related to applications for drugs for rare diseases & conditions
• Calls on the FDA to be required to develop an annual report on progress of rare disease drug applications
• Requires FDA host a public meeting to address approaches to increasing and improving engagement with rare disease or condition patients, groups representing such patients, rare disease or condition experts, and experts on small population studies, in order to improve the understanding with respect to rare diseases or conditions in terms of patient burdens, treatment options and side effects
• Directs a review of the European Union’s best practices for approving rare disease drugs

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