# A standard for tumour-agnostic approvals: minimum histologies and hierarchical modelling

Source: https://onco.cc/ideas/idea-tr1-tumour-agnostic-approval-standard/  
OnCo record `idea-tr1-tumour-agnostic-approval-standard` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- 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.
- Proposed 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

## Sources

- 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): https://doi.org/10.1136/bmj.j4530

## Connected records

- targets: [BRAF](https://onco.cc/targets/braf/), [NTRK](https://onco.cc/targets/ntrk/), [RET](https://onco.cc/targets/ret/)
- terms: [Basket, umbrella, and platform trials](https://onco.cc/terms/basket-umbrella-platform/), [Gene fusion](https://onco.cc/terms/gene-fusion/), [Microsatellite instability (MSI-H) / mismatch repair deficiency (dMMR)](https://onco.cc/terms/msi/), [Tumour-agnostic (tissue-agnostic) approval](https://onco.cc/terms/tumour-agnostic/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/), [Regulatory divergence between regions](https://onco.cc/bottlenecks/b-regulatory-fragmentation/), [Trial design, endpoints and cost](https://onco.cc/bottlenecks/b-trial-design/)
- key papers: [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](https://onco.cc/key-papers/paper-davis-bmj/)

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