Clinical DevelopmentLast reviewed April 2026

Basket Trial(Basket Trial)

A clinical trial design that tests one investigational therapy across multiple indications or tumor types sharing a common molecular target or biomarker.

Usage Examples

The basket trial enrolled patients with NTRK-fusion-positive tumors across 12 histologies, supporting tissue-agnostic approval.
Histology-specific subgroup analyses identified differential response rates requiring label limitations.

What is Basket Trial?

A basket trial evaluates a single investigational therapy across multiple diseases or patient populations that share a defining molecular feature — most commonly a specific genetic alteration in oncology. Rather than running separate trials in each tumor type, the basket groups patients by the shared target and evaluates response across tumor histologies within one master protocol.

Basket trials have supported tissue-agnostic FDA approvals (larotrectinib for NTRK fusions, pembrolizumab for MSI-H tumors). The design accelerates development of targeted therapies where disease biology crosses traditional tumor-type boundaries. Regulatory acceptance depends on biological rationale for the shared target, sufficient response rates across enrolled histologies, and adequate safety characterization across populations.

Regulatory Context

This term appears most often in clinical development workflows where submission quality, regulatory evidence, and audit readiness depend on consistent language. It is commonly referenced alongside ICH E9, FDA GUIDANCE MASTER PROTOCOLS.

FDAEMAPMDA

When This Matters

The basket trial enrolled patients with NTRK-fusion-positive tumors across 12 histologies, supporting tissue-agnostic approval.
Histology-specific subgroup analyses identified differential response rates requiring label limitations.

Common Mistakes

Applying one-region clinical assumptions to global submission strategies.
Missing protocol-to-regulation traceability for pivotal studies.
Underestimating how regional guidance updates impact trial documentation.

Related Regulations

ICH E9FDA GUIDANCE MASTER PROTOCOLS

Frequently Asked Questions

By demonstrating clinically meaningful response across multiple tumor types sharing the target, a basket trial can support indication-neutral labeling. FDA requires sufficient pan-tumor activity plus adequate per-tumor-type safety and efficacy characterization.

Varies widely based on effect size and tumor types. Typically 10-50 patients per tumor-type cohort with pre-specified stopping rules for futility. Total enrollment ranges from 100-500 depending on number of cohorts and interim analyses.

Yes. Several tissue-agnostic approvals have been based on single-arm basket trials with objective response rate endpoints. FDA evaluates based on effect size, durability of response, and alignment with unmet medical need.