Innovation for Pediatric Drugs Act
Section 3 of Give Kids a Chance Act

Innovation in Pediatric Drugs Act
Section 3 of Give Kids a Chance Act

Children are not just small adults. Drugs work differently in children and must be studied specifically for their use. Yet too often, drug development still leaves children behind. Section 2 of the Give Kids a Chance Act, the Innovation in Pediatric Drugs Act will help speed therapies to children who need them—including children with pediatric cancer and other rare diseases—by making needed changes to the pediatric drug laws.

PEDIATRIC DRUG LAWS: AN OVERVIEW

The Innovation in Pediatric Drugs Act would make needed improvements to the Pediatric Research Equity Act (PREA), a law that requires the study of drugs in children. Data resultng from PREA studies are added to drug labels to give parents and providers essential information on the safety and efficacy of drugs used in children. PREA requires drug companies to study adult drug indications in children when children could benefit from pediatric studies.

In 2017, the Research to Accelerate Cures and Equity (RACE) for Children Act amended PREA so that FDA could require companies to conduct pediatric studies of new adult cancer therapies whose molecular targets are relevant to pediatric cancers. Prior to 2017, PREA in effect excluded most oncology drugs from pediatric testng because cancers were understood by their location in the body.

THE INNOVATION IN PEDIATRIC DRUGS ACT: Ensuring Drugs for Rare Diseases are Studied in Children

Providing Equal Accountability for Pediatric Study Requirements

Due dates for PREA studies are typically deferred by FDA until a date after the approval of the drug for adults. Unfortunately, FDA has no effective enforcement tools to ensure that these studies actually get completed on time—or at all. Congress tried to solve this problem in 2012. It allowed FDA to send “non-compliance letters” to companies that failed to complete their pediatric studies. Disappointingly, this did not fix the problem. According to an analysis conducted by the American Academy of Pediatrics, as of early 2021, 123 PREA non-compliance letters had been issued, yet only 41 (33%) of these instances of non-compliance had been resolved. That lead to 82 (67%) instances of non-compliance unresolved with studies still late. The average late study was 4.4 years late. Twenty-one studies were between 5-10 years late, 7 were 10-15 years late, and 3 were more than 15 years late.

FDA requirements for postmarket studies in adults can be effectively enforced, but requirements for postmarket studies in children cannot.

If a company fails to complete adult postmarket studies, FDA can penalize the company by imposing a fine but it is prohibited, by law, from applying those penalties to pediatric postmarket studies under PREA. If FDA is not given additional enforcement tools, not only will these studies required in the past not get completed, but future studies will be in jeopardy too, including pediatric cancer studies required under the RACE for Children Act, which went into effect in 2020.

The Innovation in Pediatric Drugs Act would amend PREA to give FDA the authority it needs to ensure that legally required pediatric studies are completed in a timely way.

Investing in Pediatric Studies of Older Off-Patent Drugs

The FDA incentives and requirements under BPCA and PREA work for many newer drugs, but unfortunately cannot help encourage studies of older drugs. For this reason, Congress in 2002 authorized a BPCA program at the National Institutes of Health. This program funds NIH to do studies of off-patent drugs used in children that companies cannot be incentivized or required to conduct. To date, 18 pediatric drug labels have been changed through this program. The BPCA NIH program has been flat-funded at $25 million since its original authorization in 2002. Drug studies are expensive and while the program is an efficient use of scare resources, the $25 million funding level is insufficient to meet the current needs. When accounting for biomedical research inflation, the purchasing power of the program in 2022 was only 56% of what it was in 2002.

The Innovation in Pediatric Drugs Act would provide that of the amount made available for pediatric research to each national research institute and national center for fiscal years 2025, 2026, and 2027, the Director of NIH is authorized to make available up to one percent of such amount for pediatric clinical trials pursuant to BPCA.