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Strategic roadmap for delivery of clinical trials in rare childhood central nervous system (CNS) tumours: a multi-stakeholder consensus

Lookup NU author(s): Dr Rebecca HillORCiD, Professor Simon BaileyORCiD, Dr Debbie HicksORCiD, Professor Steven CliffordORCiD

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This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).

Abstract

© The Author(s) 2026.Rare paediatric central nervous system (CNS) tumours comprise a biologically-heterogeneous group of low-incidence diseases that present significant challenges to both therapeutic development and clinical research, limiting the availability of high-quality evidence to guide clinical care. The UK3CR Children’s Cancer Research Group (CRG) CNS Tumours Subgroup convened a multidisciplinary workshop to identify research priorities and establish a strategic framework for advancing clinical trials in this setting. Four high-priority tumour groups—craniopharyngioma, choroid plexus carcinoma (CPC), very high-risk medulloblastoma (VHR-MB), and rare embryonal and sarcomatous tumours (REST), including embryonal tumour with multilayered rosettes (ETMR)—were evaluated. Key outcomes included the feasibility of a UK-led craniopharyngioma trial, the need for enhanced national CPC data collection, and support for European early-phase platform trials in VHR-MB. REST highlighted transnational regulatory complexity requiring coordinated registries and parallel trial models. Cross-cutting barriers included regulatory discordance, contracting delays, fragmented funding, pharmacovigilance differences, data-sharing constraints, and interoperable infrastructures. The workshop emphasised innovative methodologies, including Bayesian/adaptive designs, platform trials, and external control arms, to maximise efficiency in small populations, alongside strengthened patient involvement and international collaboration. Collectively, these findings define a collaborative and internationally-aligned strategic roadmap to accelerate clinical research and improve outcomes in rare paediatric CNS tumours.

Publication metadata

Author(s): Adamski J, Apps JR, Depani S, Hill RM, Pizer B, Ajithkumar T, Bailey S, Bull KS, Carceller F, Cockle JV, Dineen RA, Douglas-Pugh JA, Gains J, Hicks D, Jorgensen M, Kilday J-P, Martinez-Barbera J-P, Ritzmann TA, Thompson E, Gates S, Hargrave D, Hackshaw A, Burke GAA, Clifford SC

Publication type: Article

Publication status: Published

Journal: British Journal of Cancer

Year: 2026

Pages: Epub ahead of print

Online publication date: 22/08/2026

Acceptance date: 10/08/2026

Date deposited: 01/09/2026

ISSN (print): 0007-0920

ISSN (electronic): 1532-1827

Publisher: Springer Nature

URL: https://doi.org/10.1038/s41416-026-03589-6

DOI: 10.1038/s41416-026-03589-6

Data Access Statement: This manuscript does not contain any foundational data.

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