Pictured: Adult holding child’s hand/gorodenkoff/iStock

Wednesday, the FDA released two draft guidances that include proposed changes to the current processes for developing and approving drugs, biological products and vaccines for children.

Marjorie Zettler, executive director of clinical science at Regor Pharmaceuticals, told BioSpace that for those who have already been involved in pediatric drug development, “there are no real surprises in these guidance documents.” But for a sponsor who is just starting out, “the new guidances are very helpful in consolidating regulatory and legislative developments over the past 18 years.”

The new guidance builds on two laws already in effect: the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). Together, the drafts are intended to replace the previous draft guidance: “How to Comply with the Pediatric Research Equity Act,” released in 2005.

The PREA, originally enacted in 2003, requires companies that study a drug in adults to also conduct pediatric studies, unless the FDA grants the company a waiver or deferral. The BPCA rewards companies that voluntarily conduct pediatric studies by giving them market exclusivity.

Pharma companies are already required to submit an initial pediatric study plan (IPSP) that spells out how they plan to test each experimental drug in children. In Wednesday’s guidance, the FDA advised these companies to submit their IPSP as early as they can, or “earlier than is required,” especially if the patient population for their drug’s indication is made up of primarily children.

Zettler explained that this guidance is a continuation of the FDA’s previous efforts to prioritize pediatric drug development. She said that since 2005, the agency had released several guidance documents related to pediatric drug development with one goal in mind: to close the gap in time between drugs being approved for adults and for children.

“Most drugs prescribed for children have not been tested in children,” according to the FDA.

Thus, many doctors prescribe drugs to children off-label, so they have not been officially approved for use in children, according to the agency.

Zettler said the recommendations outlined in the new guidance vary—some of them are specific to particular indications and address the inclusion of children in adult clinical trials. Others are more general, she said, and address regulations surrounding the use of adult clinical trial data to prescribe drugs to children.

Not all of the recommendations outlined in Wednesday’s guidance documents are new. In fact, many of them are reiterations of guidance that has been published since the PREA was first released.

For example, the new guidance requires companies to conduct pediatric clinical trials for new molecularly targeted oncology drugs with relevant targets. Zettler said this requirement was originally enacted in 2020 as part of the Research to Accelerate Cures and Equity for Children Act, or the RACE Act.

“These changes are having a positive impact. For example, FDA approvals for therapies to treat childhood cancers have increased significantly over the past decade,” Zettler said.

Rosemary Scott is a content editor at BioSpace, focusing on the job market and career development for professionals in the life sciences. You can reach her at rosemary.scott@biospace.com and on LinkedIn.