Lucas Moreno, Steven G. Dubois, Nicholas Bird, Leona Knox, Donna Ludwinski, Andrew D. J. Pearson, Maja Beck-Popovic, Rochelle Bagatell

A 2035 Clinical Research Vision and Roadmap for High-Risk Neuroblastoma

This article outlines a forward-thinking strategy for high-risk neuroblastoma. Although some progress has been made—particularly with the introduction of immunotherapy—the overall survival rates for these patients remain disappointingly low, and those who survive often endure serious long-term consequences.

The authors present a global, coordinated plan to improve outcomes by 2035. They examine why progress has been so slow, identifying critical challenges such as delayed clinical trial timelines, limited access to new treatments, and a lack of incentives for pharmaceutical companies to invest in pediatric drug development. They emphasise not only the scientific innovations needed but also the systemic, policy, and ethical shifts required to truly transform care.

The paper proposes a roadmap that calls for:

• Better and wider global collaboration

• Smarter and faster trial and adaptive trial designs.

• Integration of novel biomarkers

• More regulatory flexibility

• Expanded patient access worldwide

Ultimately, this vision is not just about improving survival rates—it’s about changing how we approach pediatric cancer research and treatment as a whole: with urgency, equity, and humanity.

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