What is a pediatric label change?

A pediatric label change refers to any update to the information on a medication’s labeling that provides specific instructions about its use. This includes updates on dosage, safety, and efficacy, particularly for special populations like children. PTN’s research generates the data needed to revise drug labels to include accurate pediatric dosing and safety guidelines, ensuring medications are used appropriately for children​.

What’s the history of PTN?

PTN was established in 2010 under the authorization of the Best Pharmaceuticals for Children Act (BPCA). PTN’s primary goal is to close the knowledge gap in pediatric medicine by conducting clinical trials that provide critical information about the dosing, safety, and efficacy of medications in children.

How long does it take to study a medicine to determine safe and effective dosages for children?

There is no standard time frame, but the most recent studies that resulted in label changes took approximately seven to nine years. The process involves several steps, including protocol development, study start-up, enrollment, data analysis, and the Food and Drug Administration (FDA) review.

What does off-label mean?

Off-label use refers to prescribing a medication in a way that differs from the FDA-approved guidelines, such as for a specific age group or condition. Many medications prescribed to children are used off-label because there is insufficient pediatric-specific research available at the time of the drug’s original approval.

What does off-patent mean?

An off-patent drug is a medication whose patent protection has expired, allowing multiple manufacturers to produce generic versions of the drug. PTN often studies off-patent drugs to ensure they have safe and effective dosing guidelines for pediatric use​.

What ages are included in pediatric trials?

PTN’s pediatric trials typically include a wide age range, from premature newborns to adolescents, as well as pregnant and breastfeeding mothers. The specific age groups vary depending on the medication and the focus of the study.

Why is pediatric dosing not included in the original Food and Drug Administration (FDA) label approval process?

Many medications are initially tested on adults, and their dosing recommendations are based on this data. Children’s unique physiology—such as their growth, metabolism, and organ function—means that adult dosing guidelines cannot be identically applied to pediatric populations. PTN conducts pediatric-specific research to close this gap​.

What safeguards are in place to protect the safety of children enrolled in PTN studies?

PTN follows strict safety protocols, including obtaining voluntary consent, disclosing all procedures, and adhering to confidentiality policies. Children are monitored closely, and safety events are reviewed by a monitoring committee, which can stop a study if harm is detected. These safeguards ensure the safety of participants throughout the study.​

How are medications selected for research by PTN?

Medications are selected through a prioritization process established by the BPCA. This process is transparent and includes stakeholder input. Each year, PTN reviews and scores potential priority areas to determine which medications will be studied.

What types of clinical trials does PTN conduct?

PTN conducts a range of clinical trials, including randomized controlled trials and observational studies, which gather robust data on pediatric medications, including off-patent and off-label drugs.

What happens if a medication studied by PTN doesn’t result in a label change?

If a medication does not result in an FDA-approved label change, PTN continues working closely with the FDA and may need to conduct further research to address gaps in data or safety concerns.

How does PTN work with the Food & Drug Administration (FDA) to ensure pediatric medications are safe?

PTN collaborates with the FDA by submitting comprehensive research data demonstrating the safety and efficacy of pediatric medications. This partnership ensures that label changes accurately reflect the best available evidence for pediatric use.

What impact has PTN had on pediatric healthcare since its inception?

Since its inception, PTN has led to 23 FDA-approved label changes, with 9 more under review. These changes provide healthcare professionals with critical information to safely prescribe medications to children.

Does PTN collaborate with international organizations on pediatric drug research?

While PTN primarily operates in the United States, it collaborates with global researchers and institutions to share data and best practices. PTN also partners with the International Children's Advocacy Network.

What role do community organizations play in PTN’s work?

Community organizations help PTN raise awareness about pediatric research and engage with families, particularly in underserved communities. These organizations play a key role in ensuring that all children benefit from safe and effective medications.​

How can parents find out about enrolling their children in PTN’s research studies?

• Parents can find information about enrolling their children in PTN’s clinical trials by visiting the PTN website or consulting with their healthcare providers. Participation in these studies helps advance pediatric care.

Where can I find more information for pregnant or breastfeeding mothers?

• PTN conducts research on medications for pregnant and breastfeeding mothers to ensure safety and effectiveness. More information can be found on PTN’s website or by consulting healthcare providers familiar with PTN’s research​.

How can I stay updated on PTN’s research and findings?

• Stay informed by subscribing to PTN’s newsletter, following their blog, or attending PTN-hosted events and webinars. Updates are regularly shared on PTN’s website and social media platforms​.