Related Experiment Video
Updated: Feb 6, 2026
08:16
Preparation and Analysis of In Vitro Three Dimensional Breast Carcinoma Surrogates
Published on: May 9, 2016
11.1K
Surrogate Endpoints in Pediatric Studies Submitted to the US FDA
Dionna J Green1, Haihao Sun2, Janelle Burnham1
1Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Clinical Pharmacology and Therapeutics
|August 11, 2018
Summary
The 21st Century Cures Act allows surrogate endpoints (SEs) to speed up drug approval. This study analyzes the use and outcomes of SEs versus clinical endpoints in pediatric drug development.
Area of Science:
- Pharmacology
- Regulatory Science
- Pediatric Medicine
Background:
- The 21st Century Cures Act (2016) aimed to expedite drug approvals.
- Surrogate endpoints (SEs) are a key provision for potentially shortening drug development timelines.
- The use and impact of SEs in pediatric trials require detailed examination.
Purpose of the Study:
- To analyze the utilization of surrogate endpoints (SEs) in pediatric drug development.
- To compare the outcomes associated with SEs and clinical endpoints in pediatric trials.
- To evaluate the role of SEs in the context of the 21st Century Cures Act.
Main Methods:
- Retrospective analysis of pediatric drug development trials.
- Identification and categorization of endpoints used (SEs vs. clinical endpoints).
- Comparison of trial success rates and approval times based on endpoint type.
Main Results:
- Data on the frequency of SE use in pediatric drug development.
- Outcomes data comparing SEs and clinical endpoints.
- Analysis of FDA approval trends related to SEs in pediatric studies.
Conclusions:
- Surrogate endpoints are increasingly utilized in pediatric drug development.
- The effectiveness of SEs in predicting clinical benefit requires ongoing evaluation.
- Further research is needed to optimize the use of SEs for pediatric drug approvals.
Related Concept Videos
01:19Precipitation Titration: Endpoint Detection Methods
6.0K
In argentometric precipitation titrations, endpoints can be detected visually by the Mohr, Volhard, and Fajans methods. In the Mohr method, adding a soluble chromate indicator gives an initial yellow color to the analyte solution. As the titrant is added, the first excess of silver ions forms a red silver chromate precipitate, marking the endpoint. The solution pH should be maintained at about 8 by adding solid CaCO3.
In the Volhard method, a standard excess of AgNO3 is first added to the...
In the Volhard method, a standard excess of AgNO3 is first added to the...
6.0K
01:26FDA Approved Drugs: Changes to Approved Drugs
273
Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...
273
01:26Pharmacokinetics in Pediatric Patients: Drug Excretion
266
In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...
266
01:17Pharmacokinetics in Pediatric Patients: Drug Distribution
314
Drug distribution in the pediatric population exhibits unique challenges and considerations due to the physiological differences between children, particularly neonates and infants, and adults. A crucial aspect of pediatric pharmacology is understanding how these differences impact the pharmacokinetics of various drugs, necessitating age-specific dosing strategies to ensure efficacy and safety.Neonates and infants have a higher total body water content, ~75%–90% of their body weight,...
314
01:24Pharmacokinetics in Pediatric Patients: Drug Metabolism
237
In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses...
237
01:23Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption
305
Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...
305