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Engel Helps Introduce Bill to Develop Treatments for Rare Childhood Dieases

Engel Helps Introduce Bill to Develop Treatments for Rare Childhood Diseases

 

Congressman Eliot L. Engel (NY-16) joined Congressmen G.K. Butterfield (D-NC) and Michael McCaul (R-TX) today in unveiling the Creating Hope Act.

 

This bipartisan legislation would permanently authorize the rare pediatric disease voucher program at the Food and Drug Administration (FDA), which has helped develop treatments for rare childhood diseases including cancers such as leukemia and neuroblastoma. Since its creation in 2012, this voucher program has helped develop 16 drugs for pediatric rare diseases.

 

“As Co-Chair of the Fragile X Caucus and a longtime advocate for research into rare diseases, this is a major priority for me. Too often, children with serious, life-threatening illnesses are left without treatment options,” said Rep. Engel. “I am pleased to join Congressmen Butterfield and McCaul on this bipartisan legislation, which will bring hope to thousands of families with children living with these challenging conditions.”

 

"Many rare diseases impact children, and over 90 percent of the approximately 7,000 known rare diseases do not have a treatment specifically targeted at the disease. The rare pediatric disease priority review voucher program aims to help incentivize drug development for our children and is, therefore, of significant importance. On behalf of children with rare diseases and their families, the National Organization for Rare Disorders is pleased to support the Creating Hope Act of 2019, which will permanently reauthorize the rare pediatric disease priority review voucher program," said Peter Saltonstall, President and CEO of the National Organization for Rare Disorders.   

 

The rare pediatric disease voucher program was last reauthorized in 2016 as part of the comprehensive biomedical legislation, the 21st Century Cures Act, which Congressman Engel helped author as a Member of the Energy and Commerce Committee. Without further Congressional action, this voucher program will expire in September 2020.

 

 

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