New FDA Guidance Explains how to Test Drugs on Pediatric Patients

A new guidance document issued by the US Food and Drug Administration (FDA) established new recommendations for how companies should conduct pediatric studies on new drug and biological products.

Testing products in pediatric patients is notoriously difficult. As FDA regulators have reiterated on numerous occasions, children "are not just small adults," and drug and biological products can behave much differently in pediatric patients compared to how they behave in adult patients.

While Congress has passed several pieces of legislation (including the Best Pharmaceutical forChildren Act and the PediatricResearch Equity Act) in the last two decades to provide both requirements and incentives for companies to test their products in pediatric patients, this legislation assumes one important fact: That companies actually know how to properly test their products in children.

FDA's latest guidance is the regulator's attempt to ensure that companies are asking all the right questions when designing their pharmacology studies.

The draft guidance, General Clinical Pharmacology Considerationsfor Pediatric Studies for Drugs and Biological Products, "is intended to assist those sponsors of new drug applications (NDAs), biologics license applications (BLAs) for therapeutic biologics, and supplements to such applications who are planning to conduct clinical studies in pediatric populations."

The guidance goes on to make specific recommendations about how sponsors should conduct pharmacokinetic (i.e. drug exposure), pharmacodynamic (i.e. drug effect) and pharmacogenetic (i.e. the effect of genes on drug effect) testing on pediatric patients, and when each type of testing is appropriate. The guidance also contains extensive information on how to design and conduct each type of testing, including the data that should be collected.

General Clinical Pharmacology Considerationsfor Pediatric Studies for Drugs and Biological Products (FR)