# Develop drugs in children first when the target is a children's target

Source: https://onco.cc/ideas/idea-bio2-paediatric-first-development/  
OnCo record `idea-bio2-paediatric-first-development` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Children wait years for drugs because adult trials come first, even when the target belongs to a childhood cancer. Some drugs should start with children.

## Summary

Paediatric oncology drug development is usually a downstream obligation, with paediatric plans triggered by adult programmes. For fusion-driven and developmental-lineage targets such as ALK in neuroblastoma, NTRK fusions, H3K27M-altered glioma and specific sarcoma fusions, the disease biology is paediatric and adult data add little. A paediatric-first pathway with matched incentives would compress timelines by years.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- Hypothesis: For targets whose prevalence is predominantly paediatric, a paediatric-first development pathway reaches approval three to five years earlier than the current adult-first sequence, without compromising safety characterisation.
- Rationale: Tumour-agnostic approvals for NTRK inhibitors and the RACE for Children Act both show that paediatric-relevant development can be accelerated when the target biology justifies it. Precocious approvals in paediatric-specific diseases exist in other therapeutic areas.
- Proposed test: Identify targets with predominantly paediatric prevalence from genomic databases, then negotiate one paediatric-first development plan with a regulator and sponsor as a demonstration case.
- Maturity: speculative
- Actor: regulator

## Sources

- Bottleneck evidence (Rare and paediatric cancers without markets): Gatta et al., Rare cancers are not so rare: the rare cancer burden in Europe (EJC 2011): https://doi.org/10.1016/j.ejca.2011.08.008

## Connected records

- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Glioma & glioblastoma](https://onco.cc/cancers/glioblastoma/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/), [Sarcomas (soft tissue, bone, GIST)](https://onco.cc/cancers/sarcoma/)
- drugs: [Dordaviprone](https://onco.cc/drugs/dordaviprone/), [Lorlatinib](https://onco.cc/drugs/lorlatinib/), [Tovorafenib](https://onco.cc/drugs/tovorafenib/)
- companies: [Children's Oncology Group (COG)](https://onco.cc/companies/childrens-oncology-group/)
- terms: [Gene fusion](https://onco.cc/terms/gene-fusion/), [H3 K27M (diffuse midline glioma)](https://onco.cc/terms/h3k27m/), [Tumour-agnostic (tissue-agnostic) approval](https://onco.cc/terms/tumour-agnostic/)
- people: [Angelika Eggert](https://onco.cc/people/angelika-eggert/), [Olivier Delattre](https://onco.cc/people/olivier-delattre/), [Stefan Pfister](https://onco.cc/people/stefan-pfister/)
- bottlenecks: [Incentives reward me-too drugs and marginal gains](https://onco.cc/bottlenecks/b-incentive-misalignment/), [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/), [Regulatory divergence between regions](https://onco.cc/bottlenecks/b-regulatory-fragmentation/)
- key papers: [Rare cancers are not so rare: the rare cancer burden in Europe](https://onco.cc/key-papers/paper-gatta-eur-j-cancer/)
- 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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