{"entity":{"id":"idea-bio2-paediatric-first-development","kind":"idea","name":"Develop drugs in children first when the target is a children's target","aka":[],"tldr":"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.","asOf":"2026-09-08","links":[{"label":"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)","url":"https://doi.org/10.1016/j.ejca.2011.08.008"}],"tags":[],"related":[],"cancers":["neuroblastoma","sarcoma","glioblastoma","all-leukemia"],"sections":[],"technologies":[],"targets":[],"drugs":["tovorafenib","dordaviprone","lorlatinib"],"companies":["childrens-oncology-group"],"institutions":[],"pathways":[],"terms":["h3k27m","gene-fusion","tumour-agnostic"],"trials":[],"people":["stefan-pfister","angelika-eggert","olivier-delattre"],"bottlenecks":["b-rare-cancers","b-regulatory-fragmentation","b-incentive-misalignment"],"keyPapers":["paper-gatta-eur-j-cancer"],"journals":[],"dependsOn":[],"notes":[],"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.","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","cost":"medium","horizonYears":7},"route":"/ideas/idea-bio2-paediatric-first-development/","neighbours":{"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"},{"id":"glioblastoma","kind":"cancer","name":"Glioma & glioblastoma","route":"/cancers/glioblastoma/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"},{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"}],"drug":[{"id":"dordaviprone","kind":"drug","name":"Dordaviprone","route":"/drugs/dordaviprone/"},{"id":"lorlatinib","kind":"drug","name":"Lorlatinib","route":"/drugs/lorlatinib/"},{"id":"tovorafenib","kind":"drug","name":"Tovorafenib","route":"/drugs/tovorafenib/"}],"company":[{"id":"childrens-oncology-group","kind":"company","name":"Children's Oncology Group (COG)","route":"/companies/childrens-oncology-group/"}],"term":[{"id":"gene-fusion","kind":"term","name":"Gene fusion","route":"/terms/gene-fusion/"},{"id":"h3k27m","kind":"term","name":"H3 K27M (diffuse midline glioma)","route":"/terms/h3k27m/"},{"id":"tumour-agnostic","kind":"term","name":"Tumour-agnostic (tissue-agnostic) approval","route":"/terms/tumour-agnostic/"}],"person":[{"id":"angelika-eggert","kind":"person","name":"Angelika Eggert","route":"/people/angelika-eggert/"},{"id":"olivier-delattre","kind":"person","name":"Olivier Delattre","route":"/people/olivier-delattre/"},{"id":"stefan-pfister","kind":"person","name":"Stefan Pfister","route":"/people/stefan-pfister/"}],"bottleneck":[{"id":"b-incentive-misalignment","kind":"bottleneck","name":"Incentives reward me-too drugs and marginal gains","route":"/bottlenecks/b-incentive-misalignment/"},{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"},{"id":"b-regulatory-fragmentation","kind":"bottleneck","name":"Regulatory divergence between regions","route":"/bottlenecks/b-regulatory-fragmentation/"}],"paper":[{"id":"paper-gatta-eur-j-cancer","kind":"paper","name":"Rare cancers are not so rare: the rare cancer burden in Europe","route":"/key-papers/paper-gatta-eur-j-cancer/"}],"roadmap":[{"id":"paediatric-oncology-roadmap","kind":"roadmap","name":"Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first","route":"/roadmaps/paediatric-oncology-roadmap/"}]}}