House Committee Passes McCaul-Butterfield 'Creating Hope Act' Incentivizes Drug Companies to Develop New Treatments for Children with Rare Pediatric Diseases

WASHINGTON, D.C.— The House Energy and Commerce Committee has approved a provision authored by Congressmen Michael McCaul (R-TX) and G.K. Butterfield (D-NC) that would incentivize pharmaceutical companies to develop new drugs for children with rare pediatric diseases, such as childhood cancers and sickle cell disease. H.R. 3059, The Creating Hope Act of 2011, was passed as part of today’s markup of a bill to reauthorize various Food and Drug Administration (FDA) user fee programs for prescription drugs and medical devices.

“This legislation will give hope to countless numbers of children and their families who are unable to treat their disease because of a lack of adequate treatments,” said Congressman McCaul, founder and chairman of the bipartisan Congressional Childhood Cancer Caucus.

“TheCreating Hope Act offers the best chance of encouraging pharmaceutical companies to develop treatments for children at no cost to taxpayers. I thank Chairman Upton and his staff for working with us to support our nation’s most vulnerable children. I look forward to a vote in the full House.”

"Children living with these life threatening conditions need access to newly developed drugs that can treat these rare diseases," said Congressman Butterfield. "Whether a disease is rare or common, the need for effective care and potential cures is the same. The Creating Hope Act provides a much-needed incentive to promote research and development that will ultimately save lives. This could not have happened without the hard work of my colleague Congressman Michael McCaul as well as Chairman Fred Upton and Ranking Member Henry Waxman of the House Energy and Commerce Committee."

The Creating Hope Act would expand the cost-neutral Food and Drug Administration priority review voucher (PRV) program, allowing pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric 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.??

The Creating Hope Act will:

• Expand the Priority Review voucher program to include treatments for pediatric rare diseases, such as childhood cancers.

• Close a loophole in current law to prevent companies from receiving a voucher for products that they already market in other countries.

• Offer unlimited transferability of vouchers to create a more easily traded asset.

• Provide greater certainty to sponsors that the drug, if approved, will qualify for a voucher, by permitting them to seek a designation from FDA before they submit their new drug application.

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