Last Updated on November 14, 2025 by

The FDA Pediatric Research Equity Act (PREA) is a law that makes pediatric studies a must for some new drugs and biologic products. This ensures that medicines are appropriately labeled for kids, helping in the growth of pediatric drug development. It covers new active ingredients, new uses, new forms, or new ways to take the medicine.

Thanks to this law, more than 500 products have been checked for kids. This has greatly improved how safe and effective drugs are for children all over the world.

Key Takeaways

• The FDA PREA mandates pediatric studies for new drugs and biologic products.
• The law applies to new active ingredients, indications, dosage forms, or routes of administration.
• Over 500 products have undergone pediatric assessment under PREA.
• The law improves drug safety and effectiveness for children worldwide.
• Pediatric drug development has been significantly advanced.

Understanding the FDA Pediatric Research Equity Act (PREA)

The Pediatric Research Equity Act (PREA) is key in making sure new drugs are safe for kids. It has greatly shaped how drugs are developed for children.

Definition and Core Purpose

PREA is a law that makes sure some drugs are tested in kids. Its core purpose is to make sure these medicines work well for children. It aims to fill the knowledge gap about how drugs affect kids.

This law is very important for kids’ health. It has changed over time to better meet kids’ needs. PREA makes sure drugs are tested in children to find any risks or differences in how they work compared to adults.

Historical Development of Pediatric Drug Regulations

Pediatric drug rules have changed a lot, with PREA being a big step. The need for special drug testing in kids was clear because there was little data. PREA was made to fix this problem.

The making of PREA was influenced by the fact that kids are not just small adults. They have special body features that can change how drugs work. The historical context of these rules shows how far we’ve come in making sure kids get safe treatments.

PREA has been updated several times to make it stronger and better for kids’ drug development. It remains a key part of the pharmaceutical industry’s focus on pediatric research.

How PREA Impacts Pharmaceutical Development

The FDA’s Pediatric Research Equity Act (PREA) has changed how drugs are developed. It makes sure kids are included in research. This law has made a big difference in drug development.

Mandatory Pediatric Study Requirements

PREA says companies must do studies on kids for certain drugs. These studies help understand how drugs work in kids. Kids and adults are different, so this is important.

Pediatric study requirements under PREA make sure drugs are safe for kids. They check the right dose, side effects, and how well the drug works in different ages.

Products Subject to PREA Regulations

PREA covers many products, like new drugs and changes to existing ones. This includes new uses, new forms, or new ways to take a drug.

• New molecular entities or active ingredients
• New indications for existing drugs
• Changes in dosage form or route of administration

Submission Processes and Documentation

Companies must send in detailed reports and documents. This includes plans, updates, and final results of studies.

The process has several parts:

1. Initial Pediatric Study Plan (iPSP) submission
2. Ongoing study reports and amendments
3. Final study results and reports

Following PREA rules is key to avoid delays or fines. Working with experts is important to handle the complex research and submission steps.

The Four Pediatric Subpopulations Under PREA

PREA identifies four key groups in pediatric care. These are neonates (birth“27 days), infants (28 days“23 months), children (2“11 years), and adolescents (12“16/17 years). Knowing these groups is vital for pediatric research.

Neonates (Birth“27 Days): Special Considerations

Neonates are very young and need special care in pediatric research. Their bodies are not fully developed, which can lead to drug side effects. It’s important to design research carefully to keep them safe while learning about drug safety.

Infants (28 Days“23 Months): Research Approaches

Infants grow and change fast. Research approaches must reflect these changes. It’s important to study how drugs work in infants to keep them safe.

Children (2“11 Years): Study Design Elements

Children are diverse and change a lot during this age. Study design elements need to account for these changes. Age, weight, and developmental stage are key to making research safe and effective.

Adolescents (12“16/17 Years): Transition Considerations

Adolescents are moving from childhood to adulthood. Research must reflect these changes. It’s important to understand how drugs affect them differently than younger or older people.

The PREA rules highlight the need to focus on these groups in pediatric research. By understanding each group’s needs, researchers can create better studies. This helps improve healthcare for kids of all ages.

PREA Waivers and Deferrals: Exceptions to the Rule

PREA has rules for pediatric studies, but there are exceptions. These include waivers and deferrals. They help deal with the challenges of studying kids.

Scientific Justifications for Waivers

The FDA gives waivers for strong scientific reasons. For example, if a drug doesn’t work the same way in kids, or if kids don’t get the disease, a waiver might be given.

Examples of scientific justifications include when a drug’s effects in kids are similar to those in adults. This means adult studies can be enough to understand its effects in kids.

Ethical Considerations in Pediatric Research

When deciding on waivers or deferrals, ethics are key. Research on kids must be done carefully to protect them.

The ethical implications of including kids in studies are weighed against the study’s benefits. This ensures kids are not put at risk.

Logistical Challenges Leading to Deferrals

Deferrals happen due to practical issues that make it hard to finish pediatric studies on time. These issues include finding enough kids to study or needing more time to make kid-friendly versions of drugs.

• Difficulty in enrolling pediatric patients
• Need for additional time to develop pediatric formulations
• Complexity of studying certain conditions in children

Application Process and FDA Review Procedures

To get waivers or deferrals, you must apply to the FDA with detailed reasons. The FDA checks these reasons carefully to make sure they meet the rules.

The FDA’s review process aims to find a balance. It considers the need for research on kids and the challenges of doing it.

PREA and BPCA: Complementary Approaches to Pediatric Research

The Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) work together. PREA sets a mandatory framework for pediatric studies. BPCA offers a voluntary framework with incentives for more research.

Comparing Mandatory vs. Voluntary Frameworks

PREA requires certain drug applications to include pediatric assessments. BPCA, on the other hand, is voluntary but offers incentives. This difference affects how drugs are studied in kids.

PREA ensures a wide range of drugs are studied in kids. BPCA’s voluntary nature allows for more flexibility. Together, they offer a strong approach to pediatric research.

Products Studied Under Both Programs

Some products are studied under both PREA and BPCA. This shows the complexity of pediatric research. For example, some drugs may need to follow PREA but also get extra study under BPCA.

• Drugs studied under PREA often have new ingredients or changes in formula.
• BPCA allows for studying both on-patent and off-patent drugs.
• The overlap between PREA and BPCA ensures a wide range of pediatric subpopulations are studied.

Exclusivity Incentives and Market Impact

BPCA offers exclusivity incentives for pediatric studies. These incentives can greatly impact the market. They give a valuable period of exclusivity.

1. The exclusivity incentives under BPCA encourage more pediatric research.
2. This leads to more drugs being labeled for pediatric use, improving treatment options for children.
3. The market impact of these incentives is significant, affecting how companies develop and market drugs.

In conclusion, PREA and BPCA have greatly advanced pediatric research. Understanding their strengths and weaknesses helps us appreciate the complexities of pediatric drug development. Continued legislative support is key.

Conclusion: The Evolving Landscape of Pediatric Drug Development

The world of pediatric drug development is always changing. This is thanks to new research, updates in rules, and the need for safe medicines for kids. The FDA had a big meeting in 2025 to get feedback for its report on kids’ drug development. This shows how hard people are working to make research and labels better for kids under the Pediatric Research Equity Act (P.R.E.A).

The P.R.E.A has been key in making sure new drugs are tested on kids when needed. This has helped doctors give better care by having accurate labels and dosages. Adding ped research to drug making has been a big step forward.

Looking ahead, new ways to study kids might include real-world data and models. The aim is to give safe and effective treatments to all kids, from babies to teens. It’s important to make sure these treatments are well-tested, including using prea animal models when it matters.

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References

1. FDA. (2012, September 30). Best Pharmaceuticals for Children Act and Pediatric Research Equity Act. StatPearls. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3470432/