Incentives for Drug Companies to Develop New Treatments for Children with Rare Pediatric Diseases Nears Reality

WASHINGTON, D.C.— The U.S. House of Representatives tonight approved a bipartisan 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 withrare pediatric diseases, such as childhood cancers and sickle cell disease.  H.R. 3059, The Creating Hope Act of 2011, was passed as part of H.R. 5651, which 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, founder and chairman of the bipartisan Congressional Childhood Cancer Caucus.  Countless numbers of children and their families who are unable to treat their disease because of a lack of adequate treatments are counting on this legislation, as will many more children who may one day learn that they have a life-threatening illness.  The Creating Hope Act offers the best chance of encouraging pharmaceutical companies to develop treatments for children at no cost to taxpayers.”

"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.  I thank my colleagues Congressman Michael McCaul, Chairman Fred Upton and Ranking Member Henry Waxman of the House Energy and Commerce Committee for their commitment to passing this bill.”

The Creating Hope Act would expand the cost-neutral Food and Drug Administration priority review voucher (PRV) program, allowing pharmaceutical companies toexpedite 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 forproducts 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.

Last week, the M.D. Anderson Cancer Center’s Chaplaincy Fund presented awards to Congressman McCaul and his wife Linda for their efforts to fight childhood cancer.  M.D. Anderson is an advocate of The Creating Hope Act.  Mrs. McCaul is a member of the Board of Visitors at M.D. Anderson, where she is Chair of theChildren's Cancer Hospital Advisory Group and also leads its Federal Relations Subcommittee. 

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Congressman and Linda McCaul with Dr. Genie Kleinermann (left) and her husband Leonard Zwelling, both of M.D. Anderson Cancer Center, at the Chaplaincy Fund Loving Hearts & Caring Hands dinner in Houston May 22.