{"entity":{"id":"idea-tr1-tumour-agnostic-approval-standard","kind":"idea","name":"A standard for tumour-agnostic approvals: minimum histologies and hierarchical modelling","aka":[],"tldr":"Some drugs are approved for any cancer with a particular mutation. Clear rules on how many cancer types must be tested, and how to combine results across them, would make these approvals more consistent and faster.","summary":"Regulators publish a standard for tissue-agnostic evidence: a minimum number of histologies with a minimum number of evaluable patients each, a Bayesian hierarchical model for borrowing across histologies with pre-specified heterogeneity thresholds, and rules for excluding histologies that show no activity. Sponsors design basket trials to the standard, and approvals list the histologies supporting them.","asOf":"2026-09-08","links":[{"label":"Bottleneck evidence (Trial design, endpoints and cost): Davis et al., Availability of evidence of benefits on survival and quality of life of cancer drugs approved by EMA 2009-13 (BMJ 2017)","url":"https://doi.org/10.1136/bmj.j4530"}],"tags":[],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["ntrk","braf","ret"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["tumour-agnostic","basket-umbrella-platform","msi","gene-fusion"],"trials":[],"people":[],"bottlenecks":["b-trial-design","b-rare-cancers","b-regulatory-fragmentation"],"keyPapers":["paper-davis-bmj"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"A published standard will reduce time to tumour-agnostic approval for genuinely histology-independent effects and prevent extension to histologies where effect is absent.","rationale":"Existing tumour-agnostic approvals (MSI-high, NTRK, BRAF V600E, RET) were reviewed case by case; basket trial statistics for cross-histology borrowing are mature and would benefit from regulatory commitment.","test":"Apply the draft standard retrospectively to past tumour-agnostic submissions and check consistency of decisions; then prospectively for the next three submissions.","maturity":"speculative","actor":"regulator","cost":"small","horizonYears":2},"route":"/ideas/idea-tr1-tumour-agnostic-approval-standard/","neighbours":{"target":[{"id":"braf","kind":"target","name":"BRAF","route":"/targets/braf/"},{"id":"ntrk","kind":"target","name":"NTRK","route":"/targets/ntrk/"},{"id":"ret","kind":"target","name":"RET","route":"/targets/ret/"}],"term":[{"id":"basket-umbrella-platform","kind":"term","name":"Basket, umbrella, and platform trials","route":"/terms/basket-umbrella-platform/"},{"id":"gene-fusion","kind":"term","name":"Gene fusion","route":"/terms/gene-fusion/"},{"id":"msi","kind":"term","name":"Microsatellite instability (MSI-H) / mismatch repair deficiency (dMMR)","route":"/terms/msi/"},{"id":"tumour-agnostic","kind":"term","name":"Tumour-agnostic (tissue-agnostic) approval","route":"/terms/tumour-agnostic/"}],"bottleneck":[{"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/"},{"id":"b-trial-design","kind":"bottleneck","name":"Trial design, endpoints and cost","route":"/bottlenecks/b-trial-design/"}],"paper":[{"id":"paper-davis-bmj","kind":"paper","name":"Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13","route":"/key-papers/paper-davis-bmj/"}]}}