Bill Includes Language Proposed by Children's Cause for Cancer Advocacy
The Children’s Cause for Cancer Advocacy (CCCA) applauds yesterday’s passage of the Food and Drug Administration (FDA) Reform Act of 2012 (H.R. 5651) by the House of Representatives with a 387-5 vote. This bipartisan legislation contains several provisions that may benefit children with cancer. The Senate passed its version of the legislation last week by a 96-1 vote.
“These actions mean that our kids once again have a place at the table – the special needs of children with cancer will be given the special attention they need and deserve,” said Maureen Lilly, Executive Director of CCCA.
A major piece of the legislation is the reauthorization of the Prescription Drug User Fee Act (PDUFA), which is set to expire on September 30, 2012. Two important components of the Food Safety and Innovation bill are the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). These pediatric provisions offer an incentive and a requirement for drugs to be evaluated for use in children. They have been the focus of intense CCCA activity in an effort to make these laws work better for children with cancer.
The legislation includes language proposed by CCCA calling for evaluation of the impact of BPCA and PREA on pediatric cancer therapies. CCCA worked with Congressional leaders on a provision in the House version to call for an FDA public meeting to examine how effective BPCA and PREA are for children with cancer. The legislation also mandates an internal FDA report to analyze the impact of these laws for children with cancer. Modifications to the programs resulting from this study will be used to improve how new therapies for children with cancer are developed. The Senate version substitutes “pediatric rare diseases” for “childhood cancer.” CCCA strongly supports the focused language on childhood cancer during the upcoming discussions to work out the differences between the two bills.
The legislation passed by both chambers also includes provisions to combat the nation’s drug shortage crisis. The provisions would mandate an early notification system that is currently voluntary, requiring drug manufacturers to notify the FDA of anticipated shortages at least six months in advance or as soon as practical. Since last November, voluntary early reporting to the FDA has prevented 128 shortages.
The legislation also establishes a taskforce to examine the causes of shortages and provide recommendations on how to prevent or alleviate them, and provides the FDA with greater authority in prevention measures.
Pediatric Rare Disease Pilot Voucher Program
A pilot program proposed in the House version of the new FDA bill would reward sponsoring companies with a transferable voucher that could be used to speed FDA review of any new drug. A company must first get approval for a brand new compound that can be used to treat only a pediatric rare disease. For this incentive, companies will also be required to pay FDA user fees to cover the cost of the pilot.
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Lawmakers are expected to convene early in June to reconcile differences between House and Senate versions and have expressed optimism that the final legislation will be ready for President Obama’s signature by the Fourth of July. CCCA will continue to work with lawmakers during resolution to ensure that the new FDA law can help children with cancer. We will keep you updated as the legislation inches closer to signage into public law.
CCCA commends Representative Anna Eshoo (D-CA), Representative Mike Rogers (R-MI) and Senator Jack Reed (D-RI) and their staffs for their hard work on the reauthorization and their interest in ensuring that it represents the needs of children with cancer. Advocates across the country have shared their stories with their legislative representatives and leaders in the pediatric oncology research community have shared their expertise and enthusiasm. Partners in advocating for this important legislation include the Alliance for Childhood Cancer, American Childhood Cancer Organization, Cancer Leadership Council, CureSearch, Leukemia & Lymphoma Society, Rally Foundation, and St. Baldrick's Foundation.
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CCCA is a leading national advocacy organization that works to achieve access to less toxic and more effective therapies, expand resources for treatment, and address the needs and challenges of childhood cancer survivors and their families. CCCA works to ensure that these needs and perspectives are integrated into the highest deliberations of cancer policy.