FDA Releases Draft Guidance on Assessing Growth, Puberty in Pediatric Trials
Trials of drugs for pediatric use should train clinical investigators and trial staff to measure and evaluate the drug’s potential impact on participants’ physical growth and sexual development, according to recent FDA draft guidance.
The recently published 10-page draft guidance recommends that trial protocols include procedures for determining a participant’s age, obtaining growth measurements, instrument calibration and evaluation of pubertal development.
“Pediatric clinical trials should include accurate, serial measurements and recordings of growth parameters if an investigational drug has the potential to affect growth or pubertal development,” continued the guidance, noting that usually “growth is assessed using measurements of weight and linear growth (length and height), and when appropriate, head circumference.”
In addition, said the guidance, sponsors should record growth measurements for a “minimum trial duration” of 12 months or discuss alternative trial durations with the necessary FDA review division.
The guidance also recommended keeping pediatric participants who stop treatment within the trial to collect growth measurements that may be needed to ensure the reliability and interpretability of analyses and results.
Trial sponsors should use a sexual maturity rating to evaluate and document pubertal development at baseline and during “regular intervals based on the potential safety concerns associated with the drug and the pubertal development stage of the pediatric participant,” the agency said.
The guidance focuses on data to support a drug’s safety and does not address the use of growth or pubertal development data on efficacy in growth disorders, the agency said, adding that sponsors should discuss how to establish efficacy with the appropriate FDA review division.
Comments on the draft guidance should be submitted by Jan. 1, 2023.
Read the draft guidancehere: https://bit.ly/3gZfPrQ.