MedPath

Pediatric Drug Testing Under PREA: An Analysis of Drugs Approved 2015-2021

• A recent study evaluates the impact of the Pediatric Research Equity Act (PREA) on drugs approved between 2015 and 2021, revealing the extent of pediatric testing. • The analysis highlights that while PREA mandates pediatric testing, exemptions and deferrals are common, impacting the availability of pediatric-specific drug information. • Congress eliminated the rare disease exemption for certain adult cancer drugs submitted after 2020, aiming to enhance pediatric testing in oncology. • The findings underscore the ongoing need to ensure children are not therapeutic afterthoughts and have access to appropriately tested medications.

Since its enactment in 2003, the Pediatric Research Equity Act (PREA) has aimed to ensure that new drugs are adequately tested in children before entering the market. A recent analysis examines the implementation of PREA for drugs approved between 2015 and 2021, shedding light on the prevalence and nature of pediatric testing during this period.

PREA's Impact on Pediatric Drug Development

PREA mandates preapproval pediatric testing for new drugs, intending to prevent children from being “therapeutic afterthoughts.” However, the law includes exemptions for drugs indicated for rare diseases, allows deferred trials for drugs otherwise ready for use in adults, and permits trial waivers in impractical circumstances, such as diseases not affecting children. Congress eliminated the rare disease exemption for certain adult cancer drugs submitted after 2020.
The study underscores the importance of ongoing evaluation of PREA's effectiveness in ensuring that children benefit from evidence-based medicine tailored to their unique physiological needs. Further research and policy adjustments may be necessary to optimize pediatric drug development and address unmet medical needs in this vulnerable population.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Recent Advances in Medical Treatments and Regulatory Approvals

The past months have seen significant progress in medical treatments and regulatory approvals, including new therapies for Alzheimer's disease, bladder cancer, and atopic dermatitis, as well as updates on rare disease innovation and biosimilar products.

FDA Grants Rare Pediatric Disease Designation to 101-PGC-005 for Systemic Juvenile Idiopathic Arthritis

The FDA has granted Rare Pediatric Disease Designation to PIF Partners' 101-PGC-005 for treating systemic juvenile idiopathic arthritis (sJIA) flares, a severe autoinflammatory condition in children.
101-PGC-005, a Type IA prodrug of dexamethasone, targets CD206+ macrophages and is currently in Phase 3 trials in India for COVID-19-induced acute respiratory distress syndrome.

Promising Pathway Act 2.0 Aims to Expedite Rare Disease Drug Approvals

The Promising Pathway Act (PPA) 2.0, under consideration in both the US House and Senate, seeks to establish a time-limited conditional approval pathway for drugs targeting rare, rapidly progressive diseases.
The proposed legislation would allow the FDA to grant initial conditional approval for two years, renewable up to eight years, for eligible drugs addressing substantial unmet needs.

FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Six Gene Therapies

The FDA has granted Rare Pediatric Disease (RPD) and Orphan Drug Designations (ODD) to six investigational gene therapies developed under the Accelerating Medicines Partnership (AMP) Bespoke Gene Therapy Consortium (BGTC).
These designations aim to incentivize the development of gene therapies for rare diseases affecting children, offering financial benefits such as Priority Review Vouchers and tax credits.

Limited Pediatric Testing for Newly Approved Drugs: A Review of PREA's Impact

A recent study reveals that only 10% of drugs approved between 2015 and 2021 completed pediatric testing before FDA approval, raising concerns about data availability for children.
Rare disease designations significantly limit the scope of the Pediatric Research Equity Act (PREA), with nearly half of new drugs being exempt from pediatric trials.

FDA Authorizes Phase 2a Trial of Progerinin for Hutchinson-Gilford Progeria Syndrome

The FDA has authorized a Phase 2a clinical trial for Progerinin, a novel drug developed by PRG Science & Technology for Progeria treatment.
The trial will evaluate Progerinin in combination with Zokinvy, the current standard of care, in 10 patients with Progeria and Progeroid Laminopathies.

Give Kids a Chance Act Aims to Accelerate Pediatric Drug Development

The Give Kids a Chance Act (H.R. 3433) has unanimously passed the House of Representatives, marking a significant step in addressing childhood cancer and rare diseases.
The Act reauthorizes the FDA’s pediatric priority voucher program for five years, incentivizing the development of innovative drugs for children.

RACE Act Drives Pediatric Cancer Drug Testing via PDX Models

The RACE for Children Act mandates testing of new oncology drugs in children if their molecular targets are relevant to pediatric cancers, impacting drug development.
Patient-derived xenograft (PDX) models, which preserve the heterogeneity of original tumors, are crucial for preclinical testing of drugs for rare pediatric cancers.

RACE Act Significantly Increases Pediatric Testing for Cancer Drugs

A recent study reveals that the Research to Accelerate Cures and Equity (RACE) Act has led to a significant increase in post-approval pediatric testing requirements for cancer drugs.
Before the RACE Act, none of the cancer drugs approved between 2017 and 2020 were subjected to mandatory post-approval pediatric testing.

FDA Expands Access to Investigational Drugs for Treatment Use

The FDA has finalized a rule to expand access to investigational drugs for patients with serious or immediately life-threatening diseases or conditions who have no comparable or satisfactory alternative therapy. This rule aims to increase awareness and knowledge of expanded access programs, detailing criteria, submission requirements, and safeguards for different types of expanded access.

Reference News

[1]
Testing of New Drugs Approved From 2015 to 2021 Under the US Pediatric Research Equity Act
jamanetwork.com · Oct 7, 2024

Congress passed the Pediatric Research Equity Act (PREA) in 2003 to ensure preapproval pediatric testing for new drugs, ...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy