Researching and developing new medicines for children is a priority for the biopharmaceutical industry. Despite the scientific complexity and challenges inherent to developing treatments for our youngest patients, there has never been more progress in pediatric health outcomes.
The progress we are seeing in pediatric health outcomes is in part due to the advancement in medicines that have been specifically studied in and approved for use in children. To help advance the development of pediatric medicines, Congress, in a bipartisan fashion, passed provisions that were eventually permanently reauthorized in two laws: the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). These policies work together to foster pediatric drug development, enabling biopharmaceutical companies to continue making significant investments in critical research areas for children.
The BPCA has been successful in advancing pediatric research. For example, ibrutinib was awarded BPCA pediatric exclusivity based on the sponsor’s research into use of the drug in pediatric chronic graft-versus-host disease (cGVHD) patients. After ibrutinib was initially approved in 2017 for adult patients with cGVHD who have tried and failed on at least one previous medicine, the U.S. Food and Drug Administration (FDA) issued a BPCA written request to the sponsor for studies in pediatric cGVHD patients. In August of 2022, the FDA approved the use of this medicine for the treatment of pediatric patients one year and older with cGVHD who have tried and failed on one previous medicine. This is the first approved treatment option for children under 12 with cGVHD and the only drug class of its kind (Brutons tyrosine kinase inhibitor or BTKi) for a pediatric population. This approval also includes a specific oral formulation designed for children, providing a helpful alternative and underscoring the importance of the BPCA in advancing pediatric research.
Unfortunately, the recent passage of the Inflation Reduction Act (IRA) puts the progress we’ve made towards treatments for children in the U.S. at risk, by forcing companies to make hard decisions in the disease areas to invest in. The IRA allows selection of certain medicines for price setting after only seven years of FDA approval and imposition of a set price beginning at year nine, substantially earlier than the average time of 13-14 years that companies currently have until they face generic competition for marketed products. The IRA also undermines long-standing incentives, including BPCA pediatric exclusivity, by allowing government price-setting notwithstanding any unexpired exclusivity.
In short, the IRA undermines the provisions of the BPCA that have proven so successful in advancing pediatric research. The IRA’s impact on innovation could jeopardize progress in pediatric drug development, putting at risk some of the nearly 600 medicines in development for children.
America’s biopharmaceutical research companies are proud of our work to bring new medicines to pediatric patients. Let’s continue to support a policy framework that makes that innovation possible. Our children, and their families, are counting on it.
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