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WASHINGTON, DC—Congressman Paul D. Tonko today spoke before the Energy and Commerce Subcommittee on Health to highlight his bipartisan Helping Experts Accelerate Rare Treatments (HEART) Act, legislation he introduced alongside Congressman David B. McKinley (R-WV) that advances a collection of reforms at the U.S. Food and Drug Administration (FDA) to strengthen the agency’s review and approval of drugs to treat rare and ultra-rare diseases, chiefly by ensuring patients, experts and doctors with direct expertise in rare diseases are consulted. 

Rep. 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 child is living with FCS.

“Tens of millions of Americans are currently living with a rare disease, including those in our Capital Region, yet only a handful of individuals currently have access to effective treatments,” Congressman Tonko said. “Accelerating and strengthening the process of reviewing treatments for rare diseases is pivotal delivering lifechanging access to care for patients and families. My legislation addresses this issue by ensuring experts as well as patient perspectives are at the forefront of the FDA’s rare disease work. I urge my colleagues to advance this legislation at once so that we may unlock to doors to treatment for those who need it most.”

The HEART Act:

• Requires a study on sufficiency and use of FDA mechanisms to incorporate patient and clinician perspective in FDA processes related to applications for drugs for rare diseases and 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.

To view Rep. Tonko’s questioning of experts at today’s hearing click, HERE.

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