McKinley, Tonko Introduce Bipartisan Legislation to Strengthen Review of Rare Disease Treatments
Legislation brings scientists and doctors with direct expertise into FDA review process
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:
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: