OnCo
ideasIdea

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

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.

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.

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.
What would test it
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
Who has to act
regulator
Cost to try
Medium ($1M to $50M)
Years to first evidence
7
Bottlenecks it attacks

Connected

17top

Pages like this

not linked directly; found by shared links