Tonko Offers Bipartisan Bill to Strengthen Review of Rare Disease Treatments

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Constituent-inspired legislation brings scientists and doctors with direct expertise into FDA review process

For Immediate Release, February 16, 2021
Contact: Matt Sonneborn, 202-680-8902

WASHINGTON—U.S. Representatives Paul D. Tonko (D-NY) and David McKinley (R-WV) introduced their Helping Experts Accelerate Rare Treatments (HEART) Act today, widely-supported bipartisan legislation that 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. 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 daughter is living with FCS.

“Our bipartisan legislation opens doors of hope and promising treatment for 25 to 30 million Americans who live with a rare disease,” Rep. Tonko said. “We can make a real difference for these individuals and their families by ensuring top experts and patient perspectives are front and center in FDA's rare disease work. I urge my colleagues to join us in moving this legislation forward without delay.”

“People suffering from rare diseases should have access to cutting-edge treatments that best serve their specific needs. This bill makes practical changes to the FDA’s approval process to expand access to safe and effective treatments for rare diseases and ensure patients have a voice at the table.” said Rep. McKinley. “By bringing doctors, scientists, and patients together to participate in this process, we can better serve the needs of the rare disease community.”

The HEART Act:

  • Requires FDA Rare Disease Program staff to participate in reviews for drugs to treat rare diseases

  • Directs the inclusion of experts in rare diseases on Advisory Committee panels for rare disease drugs

  • Assigns the FDA to develop an annual report on the number and progress of rare disease drug applications within each division at the agency

  • Requires that patients be consulted regarding Risk Evaluation and Mitigation Strategy (REMS) when a rare disease drug needs patient participation

  • Directs the General Accounting Office to review best practices used in the European Union process for rare disease drug approval including data from open label extension studies


This legislation is supported by the FCS Foundation, Haystack Project, and a broad coalition of other leaders in patient advocacy and treatment for rare diseases.

“As a constituent of Congressman Tonko I am so grateful for his leadership on the HEART Act,” said Melissa Goetz, Co-President of The FCS Foundation. “The FCS Foundation strongly supports this bill that will benefit both the FDA and all rare disease groups. As a mother of a child with FCS, I believe this bill will provide a promise of treatment options for her within her lifetime. We hope all members of the rare disease community including patients, clinicians, researchers, industry partners and friends of those impacted by rare disease will voice their support for The HEART Act by calling their representatives in Congress.”

“In our mission to expand treatment access and reimbursement for patients living with rare and especially ultra-rare diseases, the first and most important issue is that drugs that can provide benefit are reviewed and approved,” said  Jim Caro, CEO of Haystack Project. “We applaud the FDA for their many recent efforts to address the unique needs of the rare disease community. The easy-to-implement modifications in The HEART Act will represent a major advance in that process.”

Other organizations supporting the HEART Act include: Usher 1F Collaborative / Soft Bones / Alagille Syndrome Alliance / NBIA Disorders Association / Tuberous Sclerosis Alliance / Alpha-1 Foundation / The National Ataxia Foundation / American Porphyria Foundation / Cutaneous Lymphoma Foundation / International Foundation for CDKL5 Research / The Siegel Rare Neuroimmune Association / International Pemphigus and Pemphigoid Foundation / CDG Care / Usher Syndrome Coalition / Cure Cadasil / Global DARE Foundation / ICAN, International Cancer Advocacy Network / National Niemann-Pick Disease Foundation / International Fibrodysplasia Ossificans Progressiva Association / Kids With Heart National Assn for Children’s Heart Disorders / The FCS Foundation / National Lipid Association Foundation 

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