McCaul-Butterfield Creating Hope Act Signed by President

Incentivizes Drug Companies to Develop New Treatments for Children with Rare Pediatric Diseases

Texas Insider Report: WASHINGTON D.C. Today is a milestone for children who suffer from cancer and other rare pediatric diseases said Congressman McCaul after President Obama signed legislation into law today that included the bipartisan Creating Hope Act authored by McCaul (R-TX) and G. K. Butterfield (D-NC).  The Creating Hope Act of 2011 provides market incentives to pharmaceutical companies to develop new drugs for children with rare pediatric diseases such as childhood cancers & sickle cell.   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" McCaul said. Originally H.R. 3059 The Creating Hope Act was passed as Section 908 of S. 3187 of the Food and DrugAdministration Safety and Innovation Act.  The law takes effect in 90 days. This law will help pharmaceutical companies overcome market failures and change the way they look at developing treatments for children who currently dont have any or dont have treatment specific to their disease" said McCaul founder and chairman of the bipartisan Congressional Childhood Cancer Caucus.  I am pleased that Congress recognized the significance of the Creating Hope Act and passed it without hesitation" said Congressman Butterfield.  We are now one step closer to finding better treatments and cures for rare pediatric diseases.  I thank President Obama for signing this bill into law." This is the first time the United States Congress has provided a solution to thedire 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 Childrens Oncology Group and Professor of Pediatrics and Pharmacology at Childrens Hospital of Philadelphia.   The Creating Hope Act expands the cost-neutral Food and Drug Administrationpriority 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 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 thedifficulties associated with conducting clinical trials on this patient population. The Creating Hope Act will:

1. Expand the Priority Review Voucher program to include treatments for pediatric rare diseases such as childhood cancers.
2. Close a loophole in current law to prevent companies from receiving a voucher forproducts that they already market in other countries.
3. Offer unlimited transferability of vouchers to create a more easily traded asset.
4.  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.

The Creating Hope Act fundamentally transforms the way that drug companies look at rare pediatric diseases and compensates for market failures that have prevented any new treatment for pediatric cancer from being developed in a generation" Rep. McCaul said.