The US Food and Drug Administration (FDA) on Monday launched a three-month public consultation on an addendum to the International Council for Harmonisation's (ICH) guidance on pediatric clinical trials.

The addendum, E11(R1) Addendum: Clinical Investigation of Medicinal Products in the Pediatric Population, is intended to supplement ICH's E11 guidance by expanding  the discussion on issues related to pediatric clinical trials in light of scientific and regulatory advances since the guidance was published in 2000.

Specifically, the addendum includes sections on ethical considerations, age classification and pediatric formulations, as well as discussions on multiregional pediatric trials, extrapolation and modelling and simulation.

"This harmonized addendum will help to define the current recommendations and reduce the likelihood that substantial differences will exist among regions for the acceptance of data generated in pediatric global drug development programs and ensure timely access to medicines for children," the addendum states.

Pediatric Assent

In general, the addendum refers to the ethical principles laid out in E11 and emphasizes the "fundamental principle" that children should only be enrolled in a clinical study when it is "necessary to achieve an important public health need."

However, the addendum adds several considerations related to obtaining assent from children being enrolled in a study.

"When obtaining child assent, relevant elements of informed consent should be provided appropriate to the child's capacity to understand. Lack or absence of expression of dissent or objection must not be interpreted as assent," the addendum states.

Furthermore, the addendum states that investigators should "reassess the assent of a child in recognition of their evolving maturity and competency."

Multiregional Pediatric Trials

The addendum also lays out a series of questions for sponsors to address when considering an approach to multiregional pediatric clinical trials.

"Multiregional pediatric drug development programs face specific challenges due to regional differences in pediatric regulatory requirements, operational practicalities, and cultural expectations," the addendum states.

As such, the addendum says sponsors should focus on a "common scientific approach" in order to support efficient pediatric drug development.

"A common scientific approach, not common regional requirements, is at the cornerstone of efficient pediatric drug development and timely delivery of safe and effective medicines for children," the addendum states.

To guide that approach, the addendum says sponsors should address a series of questions about their drug development programs, including:

• What is the medical need in one or more pediatric populations that the drug could address?
• What objective(s) for the pediatric development program should be considered?
• What clinical studies and/or methodological approaches could be considered?

Addendum to ICH E11: Clinical Investigation of Medicinal Products in the Pediatric Population