Adult cancer medicines: starting trials with children

Background and method

Medicines developed and authorised for cancers in adults are often relevant to be investigated for cancers in children . Investigating potential cancer medicines in children is recommended to be started early and before authorisation .

This time relation is addressed on this page, to allow inspecting changes over the last decades and to further inform policies. In the U.S., requirements for molecularly targeted paediatric cancer investigations apply from mid-2020 and will be tracked in the future.

Information from the EU Clinical Trials Register and ClinicalTrials.Gov was used for this analysis . Paediatric trials were identified based on the protocol-related information that any of the paediatric age groups was to be included in the trial. Information on cancer medicines authorisations was retrieved from the EMA website. To reproduce the analysis, the R code is included below.

Start of trials with children in relation to when medicine was authorised in Europe

The figure shows all cancer medicines that are centrally authorised in Europe. Open circles indicate the date of the authorisation (for some active substances, there are two marketing authorisations). Closed circles indicate the start of a trial of the respective active substance that included the paediatric population (irrespective of subset, such as adolescents or broader paediatric age ranges). Green lines indicate that this trial was started at least 3 years before the marketing authorisation. A stark marks cancer medicines that are authorised for children (full list is in another post). Whether the trial is part of a paediatric development plan, is not visualised here. Hover over the symbols to see details.


U.S. Food and Drug Administration. 2018. “FDA Briefing Document - Relevant Molecular Targets in Pediatric Cancers: Applicability to Pediatric Therapeutic Investigations Required Under FDARA 2017.”
Herold, Ralf. 2018. Ctrdata: R Package to Aggregate and Analyse Information on Clinical Trials from Public Registers.
Jaffee, Elizabeth M., Chi Van Dang, David B. Agus, Brian M. Alexander, Kenneth C. Anderson, Alan Ashworth, Anna D. Barker, et al. 2017. “Future Cancer Research Priorities in the USA: A Lancet Oncology Commission.” The Lancet Oncology 18 (11): e653–706.
Pearson, Andrew D J, Stefan M Pfister, Andre Baruchel, Jean-Pierre Bourquin, Michela Casanova, Louis Chesler, François Doz, et al. 2017. “From Class Waivers to Precision Medicine in Paediatric Oncology.” The Lancet Oncology 18 (7): e394–404.

One thought on “Adult cancer medicines: starting trials with children

  1. Finally managed to update this post: The code is now fully compatible with my R package ctrdata version 1.1 (see references). Happy this worked out, even if on the last day of the year.

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