FDA vouchers expanded for pediatric drugs

August 1, 2012

Now legislation will enable FDA to provide marketing incentives for the development of new treatments for rare pediatric diseases, including childhood cancer and sickle cell anemia.

Since 1980, the US Food and Drug Administration (FDA) has approved just 1 new drug for the treatment of pediatric cancer compared with approvals for dozens of drugs for adults. Now legislation will enable FDA to provide marketing incentives for the development of new treatments for rare pediatric diseases, including childhood cancer and sickle cell anemia.

The Creating Hope Act, passed as Section 908 of Senate Bill 3187 of the Food and Drug Administration Safety and Innovation Act and signed into law by President Obama in July, expands FDA's priority review voucher program to include drugs for pediatric illnesses. It permits pharmaceutical companies to ask for a designation from FDA before they submit a new drug application and provides greater certainty that if their pediatric drug is approved, it will qualify for a voucher.

Drug companies have been hesitant to conduct clinical trials in children and fund the development of treatments for rare pediatric diseases because products are unlikely to recover the high costs of research, development, marketing, and distribution. Under the new law, companies will be able to qualify for FDA review of more profitable drugs in exchange for developing new cures for children.