Congressman Butterfield Introduces Legislation to Encourage Development of Treatments for Rare Pediatric Disease


WASHINGTON, D.C. – Congressman G. K. Butterfield (D-NC), along with Congressman Michael McCaul (R-TX), today introduced the Creating Hope Act of 2011.  The legislation is designed to promote drug development to treat rare pediatric diseases such as childhood cancers, sickle cell disease, pediatric AIDS and cystic fibrosis.

More than 30 million Americans affected by “rare” diseases (diseases which affect less than 200,000 people), the vast majority of which are children.  Despite the significant need, drug companies have been reluctant to invest in potential therapies targeted to small patient populations because they are unlikely to recover the high costs associated with research, development and distribution. Instead, resources are directed to more common medical conditions that affect larger populations where there is greater certainty of profit if a successful treatment is discovered and approved.

The Creating Hope Act of 2011 would expand the existing cost-neutral Food and Drug Administration (FDA) priority review voucher program, allowing pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases.

“We must do everything we can to encourage the development of therapies for all diseases; but especially those developed to save the lives of children,” said Congressman Butterfield.  “Whether a disease is rare or common, the need for innovative and effective treatment is the same. This legislation provides a much needed incentive to promote research and development that will ultimately save lives.”

Butterfield also used the introduction of the bill to highlight the importance of education and research related to sickle cell disease, an inherited blood disorder that disproportionately affects African Americans and Hispanics

The Creating Hope Act of 2011 will:

  • Expand the existing priority review voucher program (which was created to spur investment in treatment for neglected tropical diseases) to include treatments for rare pediatric diseases.
  • 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 by permitting them to seek a designation from FDA before they submit their new drug application that the drug, if approved, will qualify for a voucher.
  • Strengthen reporting and marketing requirements by requiring that the sponsor submit a statement of good faith intent to market the eligible drug.
  • Add Chagas disease, which is responsible for more deaths in Central and South America than every other parasite-borne disease, to FDA’s list of neglected tropical diseases.