The U.S. Senate sent a bipartisan provision, authored by Congressmen G. K. Butterfield (D-NC) and Michael McCaul (R-TX), to the President for his signature into law. H.R. 3059, The Creating Hope Act of 2011, will incentivize pharmaceutical companies to develop new drugs for children with rare pediatric diseases, such as childhood cancers and sickle cell. The Creating Hope Act was passed as Section 908 of S. 3187, which reauthorizes various Food and Drug Administration (FDA) user fee programs for prescription drugs and medical devices. The U.S. House approved the measure last week.
"We do a lot of things in Washington that don't mean a whole lot," said Congressman McCaul, founder and chairman of the bipartisan Congressional Childhood Cancer Caucus. "Every now and then we get the opportunity to really make a difference in the lives of others. The Creating Hope Act has the ability to actually save lives and hand down a better America to our children. In times of tough budgetary constraints, this legislation incentivizes pharmaceutical companies to finally start creating new cures for rare pediatric diseases with no cost to taxpayers."
"I am pleased that Congress has passed this critically important measure, the Creating Hope Act," said Congressman Butterfield. "We are one step closer to finding better treatments and cures for rare pediatric diseases. I look forward to President Obama signing this bill into law."
"This is the first time the United States Congress has provided a solution to the dire lack of drug development expressly for children with rare and deadly diseases," said Nancy Goodman, Founder of Kids Versus Cancer. "Congress has taken a historical stand."
"The passage of the Creating Hope Act marks an important milestone in efforts to find new cures and better treatments for children with cancer," said Dr. Peter Adamson, Chairman of the Children's Oncology Group and Professor of Pediatrics and Pharmacology at Children's Hospital of Philadelphia.
The Creating Hope Act expands 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 dozens 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.