FDA Reform Act of 2012 Signed into Law

by St. Baldrick's Foundation
July 17, 2012

Thanks to your advocacy efforts, the Creating Hope Act was signed into law as part of the FDA Reform Act of 2012. The passage of this act will save lives — helping kids get new and better treatments faster.


President Obama signed the Food and Drug Administration Safety and Innovation Act into law last week. This law seeks to address many of the nation’s healthcare concerns, including the shortage of new drugs for children with cancer.

Here’s what you need to know:

New incentives for development of childhood cancer drugs: There is little profit for pharmaceutical companies to create drugs for types of childhood cancers which is why only one childhood cancer drug has been approved by the FDA in the last 20 years. As a result of the Creating Hope Act, which was included in the FDA Reform Bill, these companies will now receive vouchers to expedite the review process for their more profitable drugs if researching and developing drugs for rare diseases, such as childhood cancers.

“This law will help pharmaceutical companies overcome market failures and change the way they look at developing treatments for children who currently don’t have any, or don’t have treatment specific to their disease,” said Congressman Michael McCaul, cosponsor of the bill and chairman of the bipartisan Congressional Childhood Cancer Caucus.

Reauthorization of BPCA & PREA: The Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA) work together as an incentive and requirement for drugs to be evaluated for use in children. While BPCA and PREA have made significant advancements in how medicines can be used in children, they have had a modest impact on the development of new drugs for children with cancer. The FDA Reform Act mandates an internal FDA public meeting to examine the effectiveness of BPCA and PREA on children with cancer no later than December 21, 2013. The FDA is also required to issue an internal report, including recommendations to facilitate the development of new and better therapies for children with cancer.

Creation of the FDA’s Pediatric Subcommittee of the Oncologic Drugs Advisory Committee: This will be a permanent public forum through which the agency will discuss and improve therapies for children with cancer.

Secretary of Health and Human Services required public meeting for pediatric rare diseases: At least one public meeting a year is now required for focus on encouraging and accelerating the development of new therapies for all pediatric rare diseases, including childhood cancers. (Including a strategic plan as a result of the meeting.)

Thank you to the St. Baldrick’s families and volunteers who played an important role in the passage of this bill by joining with other advocates from across the country during Childhood Cancer Action Days, and for those serving from home as “virtual advocates,” sending hundreds of messages to Congressional offices.

While much has been accomplished, our work is not done. Join the St. Baldrick’s advocacy action network, Speak Up for Kids’ Cancer, to stay informed and receive alerts when your help is needed the most.

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