McKinley, Tonko Introduce Bipartisan Legislation to Strengthen Review of Rare Disease Treatments

Legislation brings scientists and doctors with direct expertise into FDA review process

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Washington, February 16, 2021 | comments

WASHINGTON, D.C. —Today, U.S. Representatives David B. McKinley, P.E. (R-W.Va.) and Paul D. Tonko (D-NY) introduced the Helping Experts Accelerate Rare Treatments (HEART) Act. This 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.

“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.”

“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.”


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 several of the nation’s leading organizations in rare disease advocacy including the Familial Chylomicronemia Syndrome (FCS) Foundation, HAYSTACK Project, and a broad coalition of other leaders in patient advocacy and treatment for rare diseases. 

Other organizations that support 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 Association for Children’s Heart Disorders
  • National Lipid Association Foundation 


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