Packages/Pediatric dosing
Population PK with maturation · ICH E11A

Dose children with less pediatric data

Scope this package
Delivery
Up to 6 weeks from a clean dataset
Price
Fixed, quoted after the call
Typical model risk
High

What is at stake

Pediatric studies are slow to enrol and costly per patient. When disease and response are similar enough, extrapolation lets exposure matching carry much of the evidence. The dose then has to be right the first time.

Question of interest

Which doses in children achieve exposures matching adults, and how much pediatric efficacy data is needed?

A population PK model with allometry and maturation, built on adult data and any pediatric data available, predicts exposure by weight and age band. It supports dose selection, sampling design and the quantitative part of the extrapolation concept and plan.

What reviewers will ask

Is maturation of clearance handled for the youngest children?

Maturation functions are included for the relevant pathways, with their uncertainty shown, not assumed away.

Is the adult exposure target the right one?

The target comes from the adult exposure-response evidence and is defined before any pediatric simulation.

Will the sampling design estimate the parameters that matter?

Designs are evaluated for parameter precision before the protocol is finalized.

What you file with the agency

  • Quantitative sections of the PIP or iPSP
  • Pediatric dose rationale for the protocol and briefing document
  • Population PK report with maturation, and M15 assessment table
  • Sampling-design justification

What we need from you

  • Adult PK data and the adult exposure-response evidence
  • Any pediatric PK data, including from other indications
  • The current PIP or iPSP and the agency feedback so far
Simulated steady-state AUC by pediatric weight band compared with the adult exposure range.
Exposure matching by weight band.

What it may replace

Where the extrapolation concept holds, the analysis can reduce the size of pediatric efficacy studies or support a PK-based bridging study. The extent is agreed with the agencies.

Scientific approach

Model. Allometric scaling with fixed or estimated exponents, maturation functions for clearance pathways in young children, and covariate effects carried over from adults where justified.

Target. The adult exposure range that underpins efficacy and safety, defined before simulation.

Dosing. Weight-band or mg/kg schemes compared for the share of children within the target range, with practical constraints such as available strengths.

Design. Sampling schedules evaluated for parameter precision, with the minimum sample size the extrapolation plan needs.

Relevant guidance: ICH E11A on pediatric extrapolation; ICH E11(R1); FDA and EMA pediatric guidance; ICH M15.

Is this the decision in front of you?

Fixed price, paid in two milestones. A guaranteed date. Agency questions on this analysis answered at no extra cost. Pricing · How we work

Scope this package