# Make paediatric combination studies part of every relevant adult cancer drug approval

Source: https://onco.cc/ideas/idea-tr2-paediatric-combo-prea/  
OnCo record `idea-tr2-paediatric-combo-prea` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Children's cancers are treated with combinations, but companies study new drugs in children one at a time. Approvals should require the combination study children actually need.

## Summary

The US RACE for Children Act requires paediatric evaluation of molecularly targeted adult cancer drugs, but permits single-agent studies that rarely lead to use, because paediatric standards of care are multi-drug backbones. Requiring the paediatric plan to include a combination with the relevant backbone, designed with cooperative groups, would produce actionable evidence.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- Hypothesis: Drugs whose paediatric plans include a backbone combination will reach paediatric labelling or guideline inclusion at least twice as often within seven years as drugs studied as single agents.
- Rationale: Nearly every paediatric oncology advance came from adding an agent to an existing backbone in a cooperative-group trial, not from monotherapy.
- Proposed test: Compare paediatric labelling outcomes for agents studied in combination versus monotherapy under current paediatric plans, then pilot the requirement with two new agents.
- Maturity: speculative
- Actor: regulator

## Sources

- FDA paediatric oncology: https://www.fda.gov/about-fda/oncology-center-excellence/pediatric-oncology

## Connected records

- companies: [Children's Oncology Group (COG)](https://onco.cc/companies/childrens-oncology-group/)
- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/), [Standard-risk B-cell acute lymphoblastic leukaemia in children](https://onco.cc/cancers/all-paediatric-standard-risk/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/), [Too many combinations to test](https://onco.cc/bottlenecks/b-combination-space/)
- roadmaps: [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)

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