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Tonko, McKinley Advance Bill to Strengthen Review of Rare Disease Treatments

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Washington, July 9, 2020 | comments

WASHINGTON—U.S. Representatives Paul D. Tonko (D-NY) and David McKinley (R-WV) are introducing the Helping Experts Accelerate Rare Treatments (HEART) Act today, legislation 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, in particular by ensuring scientists and doctors with direct expertise are involved throughout the process of reviewing a treatment for a condition. 

“Rare and ultra-rare diseases present a unique challenge for drug development because, while some 25 to 30 million Americans have a rare condition, any one of these conditions by definition only impacts a relatively small number of individuals,” said Rep. Tonko. “Our bipartisan legislation tackles this issue by ensuring rare disease experts are at the table as these therapies are being reviewed by the FDA as well as amplifying the patient perspective as safety protocols are being established. As a result, millions of Americans will see new glimmers of hope as new treatments start to emerge with FDA approval and better information to guide health providers in the safe and effective use of those treatments. I urge my colleagues to join us in moving this legislation forward without delay.” 

“Often times people suffering from rare diseases have few or no treatment options available. This bill makes practical changes to FDA’s approval process that can lead to new treatments and ensure patients have a voice at the table,” said Rep. McKinley. “By having more experts available to participate in this process, we can better understand and address the needs of patients moving forward.” 

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. 

“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.”

“The HAYSTACK Project shares our commitment to finding ways to improve the regulatory review process for drugs to treat rare diseases in the U.S. and we are very pleased to join with them in supporting this important bill,”  said Melissa Goetz, Co-President of The FCS Foundation and a constituent of Congressman Tonko. “We invite all members of the rare disease community including patients, clinicians, researchers and industry partners to voice their support for The HEART Act by calling their representatives in Congress.”

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