A new multi-stakeholder consensus roadmap published in the British Journal of Cancer outlines how clinical trials should be structured for rare childhood central nervous system tumors. The document targets operational barriers that have slowed testing in low-patient-population settings and calls for more practical, inclusive trial approaches. The roadmap centers on aligning scientific study design with real-world trial conduct—addressing how sponsors, sites, and regulators can collaborate to reduce friction before first patient enrollment. For developers, the guidance is aimed at making trial timelines more achievable and improving feasibility for smaller indications where historical trial infrastructure has lagged behind scientific advances.