Thursday 22 Aug 2019

Creating Hope for Children with Rare Diseases

US Congressman G.K. Butterfield (D-NC)

The U.S. House of Representatives approved a provision authored by Congressmen Michael McCaul and G.K. Butterfield Wednesday that would provide incentives for pharmaceutical companies to develop new drugs for children with rare pediatric diseases, such as childhood cancers and sickle cell disease. The Creating Hope Act of 2011 reauthorizes various Food and Drug Administration (FDA) user fee programs for prescription drugs and medical devices. If the Senate approves the measure, it would go to the president for his signature.

“There aren’t many bills that pass the House that give instant hope to so many people in need. This one does,” said Congressman McCaul.

“Children living with these life threatening conditions need access to newly developed drugs that can treat these rare diseases,” said Congressman Butterfield. “The Creating Hope Act incentivizes pharmaceutical research and development, which will undoubtedly lead to countless young lives being spared from rare diseases.”

Since 1980, the FDA has approved only one new drug for treatment of childhood cancer, compared to 50 for adults. Despite this significant unmet medical need, pharmaceutical companies have been reluctant to develop drugs for rare pediatric diseases because it requires making an investment in products that are unlikely to cover the high costs associated with their research, development, marketing and distribution. Developing products for children is particularly challenging because of the difficulties associated with conducting clinical trials on this patient population.

 Goldsboro Daily News.  Amy Roux, Curtis Media, 2012.

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