Basket trial

A basket trial is a type of clinical trial that tests the effectiveness of a new drug or other substance in patients who have different diseases that share the same mutation or biomarker. The basket trial offers treatment that targets this specific mutation or biomarker.

While basket trials may be used to investigate patients who have any type of disease, clinical trials involving different kinds of cancer are the most likely to use this trial type. Drug development for neurodegenerative disorders (NDDs), however, has seen increasing use of basket trials since one drug can often influence multiple NDDs.

Basket trials are considered a “master” protocol design as they answer multiple questions in a single study. They are often used in the early stages when developing a new agent as they allow researchers to examine different types of cancer with the same mutation/biomarker to see which tumor types are most responsive to the therapy, providing a direction for future development.

Basket trials can help to make the drug development process more efficient by enhancing recruitment, reducing redundancy in trial completion, and using biomarkers that are relevant to the treatment’s mechanism of action.