FDA Proposes Guidance On Conducting Clinical Trials For Newborns

Federal regulators have proposed new guidelines, which are designed to make clinical trials safer for newborns, and reduce the risk of potential drug side effects.

In a draft guidance published in the Federal Register on August 1, the FDA provides guidelines for conducting clinical trials that involve newborns. The agency indicates that the effort will help the drug industry create safe clinical trials to study drugs and therapies intended for newborns. The main goal is to keep infant safety at the forefront, while developing therapies that are effective for newborns’ unique needs.

The draft guidance lays out the framework for clinical trial design for newborns, a task the drug industry often does not take for fear of safety concerns among young infants. It is the first draft guidance issued focusing on the conditions to consider in order to design a clinical trial for newborns.

“The FDA is committed to gaining more information on the safety and efficacy of existing therapies given to our youngest patients, and we also want to encourage the development of new therapies, so today we are issuing our first draft guidance regarding specific considerations on the design of clinical trials involving newborns, Dr. Susan McCune, director of the FDA’s Office of Pediatric Therapeutics, said in a July 31 press release. “Today’s draft guidance provides clarity for industry regarding clinical trial study design, drug dosing and analysis considerations.”

The neonatal period is considered the day of birth plus 27 days later, for a full-term infant. For preterm infants, the neonatal period is the day of birth through the original expected day of birth plus 27 days. These are the earliest and most vulnerable days for a newborn.

Typically, clinical trials are not conducted on newborns because they are so young and still undeveloped. The side effects often experienced during clinical trials can be too severe and risky for newborns

For that reason, in order to treat infants in need of novel medical care, healthcare professionals in the neonatal intensive care unit (NICU) often give infants drugs for uses they were not intended or designed for. They often have to do this without clinical trial data on the potential side effects. Additionally, many infants born preterm face unique challenges that should be considered to determine the correct medication and treatment, but without clinical trials those are difficult to determine.

The guidance indicates the researchers should receive input from a multidisciplinary team that includes neonatal care professionals as well as parents.

Conducting clinical trials on newborns, using the right conditions, design and a focus on safety, can help drug makers make medications and therapies that are not only effective for very young infants, but safe and with fewer side effects, McCune said.

The FDA is taking public comment on the draft guidance until October 30. Comments can be made electronically through, or through written submissions sent to Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.

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