Drug Development

Background

In the past 20 years, just two new drugs have been approved for any pediatric cancer. The small patient population of children with cancer provides little market incentive for the biopharmaceutical industry to develop new pediatric oncology drugs. Treating children with cancer has relied upon the use of drugs developed for adult cancers, which can differ substantially from childhood cancers. Congress has acknowledged obstacles to the appropriate use of all drugs in children by enacting the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), two laws that provide requirements and incentives for testing drugs in children. Together, BPCA and PREA create a “carrot and stick” approach that can yield important new safety and dosing information for pharmaceutical products used to treat children.

Legislation

Best Pharmaceuticals for Children Act (BPCA)

The Food and Drug Administration Modernization Act of 1997 (FDAMA) created a financial incentive for companies. They would be awarded six months additional market exclusivity for all uses of a drug if they conduct pediatric studies in accordance with a written request from the Food and Drug Administration (FDA). This incentive generally delays marketing of generic forms of a drug for six months and is known as “pediatric exclusivity.”

In 2002, Congress passed BPCA, which reauthorized the pediatric exclusivity incentive. Under BPCA, sponsors receive pediatric exclusivity as an incentive to conduct studies of drug and biologicals for use in children.

Pediatric Research Equity Act (PREA)

PREA was enacted in 2003, and was designed to work in conjunction with BPCA. PREA requires that pharmaceutical manufacturers conduct pediatric studies of certain drugs and biologics before they are marketed, unless FDA grants a waiver or deferral. PREA requires that pediatric studies be conducted only for the same indications (therapeutic uses) described in the drug application (e.g., adult bone cancer, childhood bone cancer). In addition, PREA specifies that studies are not required for drugs used to treat rare or orphan diseases. By definition, all childhood cancers are rare diseases, therefore PREA does not apply to all childhood cancers.

CCCA Role

BPCA and PREA are set to expire on October 1, 2012. While BPCA and PREA have yielded important new safety and labeling information for children, the laws have had a modest impact on children with cancer. Children’s Cause for Cancer Advocacy (CCCA) is committed to identifying policies that could advance the development of better, less toxic therapies for children with cancer. The reauthorization of BPCA and PREA provide a unique opportunity to engage with lawmakers to strengthen the laws to potentially advance pediatric oncology drug development. For the past year, CCCA has been analyzing and discussing with experts ways in which BCPA and PREA could be modified so that they can actually incentivize and require companies to develop new therapies for children with cancer.