What is the Pediatric Research Equity Act (PREA)?
The Pediatric Research Equity Act (PREA) is a U.S. law that requires drug and biologic sponsors to conduct pediatric studies for many medicines that are approved in adults, so that labeling can reflect how the product works (and how it should be used) in children [1]. It is part of how the FDA supports pediatric therapeutic information, rather than leaving pediatric use to extrapolation from adult data.
Which products does PREA apply to?
PREA generally applies to applications for new active ingredients and certain changes to existing drugs, including situations where the adult product is being authorized and pediatric information is expected to be needed for safe and effective use in children [1].
What does PREA require sponsors to do?
For products subject to PREA, sponsors must submit pediatric study plans to the FDA and carry out studies designed to answer key pediatric questions (such as dosing, effectiveness, and safety in relevant pediatric age groups) [1]. The FDA can also grant waivers or allow deferrals in some cases, depending on whether studies are necessary or can be timed appropriately.
What are waivers and deferrals under PREA?
A waiver means pediatric studies are not required for a product or for certain pediatric subsets. A deferral allows studies to be delayed until later stages of development, often to avoid delaying adult access when it’s not yet feasible to study children or when it would not be scientifically appropriate [1].
How does PREA differ from the Pediatric Exclusivity provision?
PREA is about requiring pediatric studies and a pediatric label update when appropriate. Pediatric exclusivity (a separate mechanism in U.S. law) can provide additional market exclusivity when sponsors complete eligible pediatric studies. PREA drives study obligations; pediatric exclusivity provides an incentive for completing them.
Why was PREA created?
PREA was designed to address a long-standing problem: many drugs reached the market with little or no pediatric evidence, leaving clinicians to rely on extrapolation or off-label use. By making pediatric assessments more systematic for eligible products, PREA aims to improve the pediatric evidence base and labeling [1].
Where can I find the official FDA guidance and program details?
FDA’s main PREA resources are organized under its pediatric drug and biologic program materials, including information on pediatric study plans, timelines, and how waivers/deferrals are handled [1].
Sources
- https://www.fda.gov/drugs/pediatric-medicines/pediatric-research-equity-act-prea