# Basket trial

Source: https://onco.cc/terms/basket-trial/  
OnCo record `basket-trial` (Term). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A basket trial tests one drug in patients whose cancers share a mutation or marker, whatever organ the cancer started in; each organ type is a basket, and the trial asks whether the drug works across them.

## Summary

The basket design turns the usual logic of a cancer trial inside out. Instead of one cancer and one drug, it takes one molecular target and enrols any cancer that carries it, sorting patients into baskets by tumour type. Each basket is usually a small single-arm cohort with response rate as its endpoint, and the trial can close baskets that show no responses while expanding the ones that do. Baskets are the natural design for rare alterations that appear in a few percent of many different cancers, where no single-cancer trial could ever recruit enough patients.

The worked example is NAVIGATE, the larotrectinib trial that enrolled adults and children with TRK fusion-positive solid tumours of any type. Pooled with the adult phase 1 and the paediatric SCOUT study, 75 percent of the first 55 patients responded across 17 tumour types, and the result supported the 2018 US accelerated approval of larotrectinib for a genomic marker regardless of tumour site, the first of its kind. ROAR was a basket trial of dabrafenib and trametinib in BRAF V600E-mutant cancers whose anaplastic thyroid cancer cohort of 36 patients showed a 56 percent response rate and produced the first targeted therapy for that disease. NCI-COG Pediatric MATCH was a screening protocol with a dozen single-agent arms: in the first 1,000 children sequenced, 31.5 percent had an actionable alteration and 13.1 percent enrolled on a treatment arm, proving that the plumbing works even though most single drugs did little.

The design has known weaknesses. Small baskets give wide confidence intervals, so a handful of responses can look like a breakthrough. A marker can be a true driver in one organ and a passenger in another, which is why BRAF inhibitors work in melanoma and hairy cell leukaemia but not in BRAF-mutant colorectal cancer without a partner drug. And a basket trial cannot show that patients live longer, because there is no control arm; the response rate and its durability are what regulators weigh, usually with a confirmatory commitment attached.

## Fields

- Kind: Term
- Last checked: 2026-09-17
- Also known as: basket trial; basket trials; basket study; basket studies; basket design; histology-agnostic trial; histology-independent; tumour-agnostic trial; tissue-agnostic trial; one drug many cancers; pan-tumour basket; pan-tumor basket; basket cohorts; cohort expansion by tumour type

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Basket_trial
- Wikipedia: https://en.wikipedia.org/wiki/Basket_trial
- Woodcock and LaVange, Master protocols to study multiple therapies, multiple diseases, or both (NEJM 2017): https://doi.org/10.1056/NEJMra1510062

## Connected records

- terms: [Accelerated approval](https://onco.cc/terms/accelerated-approval/), [Basket, umbrella, and platform trials](https://onco.cc/terms/basket-umbrella-platform/), [Bayesian trial design](https://onco.cc/terms/bayesian-trial-design/), [Enrichment and biomarker-stratified designs](https://onco.cc/terms/biomarker-stratified-design/), [Objective response rate (ORR)](https://onco.cc/terms/orr/), [Phase 1, 2 and 3 trials](https://onco.cc/terms/trial-phases/), [Seamless, adaptive and Bayesian trial designs](https://onco.cc/terms/seamless-adaptive/), [Single-arm trial](https://onco.cc/terms/single-arm/), [Tumour-agnostic (tissue-agnostic) approval](https://onco.cc/terms/tumour-agnostic/), [Umbrella trial](https://onco.cc/terms/umbrella-trial/)
- fronts: [Drug Discovery Platforms](https://onco.cc/fronts/drug-discovery/)
- drugs: [Dabrafenib](https://onco.cc/drugs/dabrafenib/), [Larotrectinib](https://onco.cc/drugs/larotrectinib/), [Pralsetinib](https://onco.cc/drugs/pralsetinib/)
- trials: [ARROW (thyroid cohorts)](https://onco.cc/trials/arrow-thyroid/), [NAVIGATE](https://onco.cc/trials/navigate/), [NCI-COG Pediatric MATCH (APEC1621)](https://onco.cc/trials/pediatric-match/), [ROAR (anaplastic thyroid cancer cohort)](https://onco.cc/trials/roar-atc/)
- bottlenecks: [Trial design, endpoints and cost](https://onco.cc/bottlenecks/b-trial-design/), [Trials enrol too few, too slowly](https://onco.cc/bottlenecks/b-trial-enrolment/)

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