FDA draft guidance aims to ensure accurate measurements of pediatric growth

Joint draft guidance from two centers at the US Food and Drug Administration (FDA) outlines ways sponsors can measure pediatric growth consistently in clinical trials for drugs used to treat both rare and common diseases.

“If an investigational drug or biological product may affect growth or pubertal development, then accurate, serial measurement and recording of growth parameters are essential for data interpretation in pediatric clinical trials,” the agency announced in a pre-publication notice in the 31 October Federal Register. FDA further noted that “identifying the onset and progression of puberty are essential for accurate interpretation of growth data.” The guidance is expected to appear on 1 November.

The guidance, issued jointly by the Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), does not address drugs used to treat growth deficiencies, precocious puberty or delayed puberty.

The guidance addresses the collection of growth measurements such as weight, linear growth, and in certain circumstances, head circumference.

Sponsors should consider developing a protocol to train investigators and trial site personnel on methods for collecting growth data and procedures for conducting physical examinations.

Companies should avoid collecting self-reported growth measurements as they may not be accurate. Measurements would only be acceptable if taken by trained health care professionals, the guidance notes.

To reduce measurement errors, sponsors should establish procedures for calibrating measuring instruments; such procedures should address how measurements are obtained from uncooperative children.

To ensure consistency in reporting chronological age, for participants ages 3 years and older, sponsors should use their chronological age to the nearest month and year. For participants younger than 3 years, sponsors should determine whether the participant was born at term (37 weeks gestation or later) or preterm.

For accurate weight assessments, sponsors should use a scale with an electronic, or digital reading that allows for adjusting the scale to zero prior to weighing subjects. All scales should be calibrated at the same time before each study visit.

To measure head circumference, sponsors should use a nonelastic tape measure, and measure the maximum diameter of the head only.

To assess pubertal development, sponsors should use a sexual maturity rating such as the Tanner staging method and evaluate development at baseline and ensure that investigators are adequately trained on these ratings. “Whenever possible, the same trial health care professional should evaluate pubertal development,” the guidance states.

FDA will give industry 60 days to comment once the notice is published. Submit comments to www.regulations.gov and refer to Docket No. FDA-2022-D-0760.

FDA notice

Draft guidance

FDA draft guidance aims to ensure accurate measurements of pediatric growth

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