{"entity":{"id":"idea-tr2-paediatric-combo-prea","kind":"idea","name":"Make paediatric combination studies part of every relevant adult cancer drug approval","aka":[],"tldr":"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.","asOf":"2026-09-08","links":[{"label":"FDA paediatric oncology","url":"https://www.fda.gov/about-fda/oncology-center-excellence/pediatric-oncology"}],"tags":[],"related":["childrens-oncology-group"],"cancers":["neuroblastoma","all-leukemia"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":["b-combination-space","b-rare-cancers"],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"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.","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","cost":"medium","horizonYears":5},"route":"/ideas/idea-tr2-paediatric-combo-prea/","neighbours":{"company":[{"id":"childrens-oncology-group","kind":"company","name":"Children's Oncology Group (COG)","route":"/companies/childrens-oncology-group/"}],"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"},{"id":"all-paediatric-standard-risk","kind":"cancer","name":"Standard-risk B-cell acute lymphoblastic leukaemia in children","route":"/cancers/all-paediatric-standard-risk/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"},{"id":"b-combination-space","kind":"bottleneck","name":"Too many combinations to test","route":"/bottlenecks/b-combination-space/"}],"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/"}]}}