Today, in their ongoing effort to continue improvements to the safety and effectiveness of medications for children, Reps. Anna G. Eshoo (D-Palo Alto), Edward J. Markey (D-Mass.), and Mike Rogers (R-Mich.) introduced H.R. 4274, legislation to reauthorize the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA). This bipartisan reauthorization will make significant improvements to the laws, which encourage companies to appropriately study medications for children.
"As the original champion for the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act, I'm proud that these laws have dramatically improved the number of drugs that have been studied and approved for children," said Rep. Eshoo. "Government and private industry have worked together to fill an important void. Today, our legislation will provide greater transparency and accountability in the programs, while improving communication between FDA and companies. We've come a long way, and I look forward to a day when we know how all medicines will affect children."
Congress first recognized the need to ensure that drugs were being studied in children in 1997 when it passed the Better Pharmaceuticals for Children Act (BPCA), a bill to incentivize the study of off-label uses in pediatric populations by offering companies an additional six months of patent life on their product. In 2003, Congress passed the Pediatric Research Equity Act (PREA) to study on-label indications in children, when safe and appropriate.
"What medications are good for the goose are not necessarily good for the gosling. Reauthorizing the laws that promote the study of pediatric drugs will ensure that children's medical needs are not relegated to the kids table and that children receive equal attention in the development of medications," said Rep. Markey.
"These two successful laws have led to180 new drugs being studied in children, and more than 400 drug labels have been updated with critical pediatric information, leading to more information about pediatric drug safety, effectiveness, dosing and formulations," said Rep. Rogers. "Drug manufacturers, physicians, patients, and the FDA all recognize that BPCA and PREA have worked incredibly well to increase the study of prescription drugs in children. Making both programs permanent provides a more certain regulatory environment for drug makers in pediatric drug research."
The reauthorization of the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act will:
Provides the Food and Drug Administration (FDA) the necessary enforcement tools to ensure that companies complete their required pediatric studies under PREA on time.
Increases transparency of the status of pediatric clinical trials required under PREA.
Ensures the timely submission of a company's "Pediatric Study Plan", a blueprint for how a company plans to study their drug in children.
Ensures that neonatologists are involved in the process of reviewing and planning pediatric clinical trials.
"BPCA and PREA have greatly improved pediatric labeling of HIV medications. This is especially important for children living with HIV, because under-dosing can lead to drug resistance, and overdosing can lead to toxicity," said Dr. Laura Guay, Vice President of Research for the Elizabeth Glaser Pediatric AIDS Foundation (EGPAF). "The bipartisan legislation introduced today strengthens these important laws to ensure that children are prioritized in the drug development process, and have access to life-saving medicines labeled especially for their unique health needs."
"We are pleased that Representatives Rogers, Eshoo and Markey have introduced legislation to reauthorize BPCA and PREA that will continue to incentivize the development of lifesaving therapies for children," said Jim Greenwood, CEO of the Biotechnology Industry Organization (BIO). "Together BPCA and PREA have been remarkably successful in generating pediatric drug information for physicians and parents, contributing to improved health outcomes for children. Making BPCA and PREA permanent will result in continued investment in pediatric research infrastructure and ensure that new drugs and biologics will be used safely and appropriately in pediatric patients."
"We applaud Representatives Mike Rogers, Anna G. Eshoo, and Edward J. Markey for their leadership in authoring the BPCA and PREA Reauthorization Act of 2012. This bipartisan legislation is critical because it both renews BPCA and PREA, two pediatric drug testing laws that have improved the health and well-being of millions of children, and it makes several important policy improvements called for by the Academy and the Institute of Medicine (IOM) in its recent Safe and Effective Medicines for Children report," said Robert W. Block, MD, FAAP, president, American Academy of Pediatrics. "Children are not just small adults. Drugs work differently in children than in adults and must be studied specifically for their use. Because of BPCA and PREA, 427 drug labels have been revised with important pediatric information. While there has been significant success, more progress is needed, and these laws must be reauthorized and strengthened by improving the timing and quality of pediatric research as well as ensuring that required studies are completed by their due date. Your legislation does just that."
"Permanent reauthorization of BPCA and PREA would provide biopharmaceutical companies with the certainty that they need in order to plan and conduct pediatric studies," said John Castellani, PhRMA's CEO. "Because these studies can take several years, it is important for companies to know that the pediatric-research programs will continue throughout the span of the trial. We thank Representative Rogers, Representative Eshoo and Representative Markey for their support for permanent reauthorization, which will provide a more-predictable regulatory path and will help to stimulate increased pediatric research."