There has been a growing emergence in the utilisation of basket studies and it’s not difficult to see why. Progress in genomics, tumour biology and statistics has led to advances in “precision oncology”. Cancers that were once viewed as homogeneous in terms of location and treatment strategy are now better understood to be increasingly heterogeneous across biomarkers and genetically determined subgroups. No two cancers are the same; tumours differ from patient to patient and few patients may noticeably improve with treatment, whilst others experience no benefit at all. As a result, we have seen a shift towards targeted agents, and it has become more common for trials to focus on a specific mutation at a particular location.
When thinking about the timeframe from initial drug discovery to regulatory review, we need to embrace novel clinical trial designs that improve efficiency. With this in mind, there has been an observable trend towards investigating multiple target-treatment pairs in parallel, either within, or across tumour types. The term “master protocol” refers to a general framework whereby multiple parallel drug studies are operated under one overarching protocol. A basket trial is a type of master protocol that tests the effect of one drug on a single mutation in a variety of tumour types. That is, they include patients with a certain genetic mutation in common regardless of the site or origin of cancer in the body, so patients have cancer at a variety of sites such as lung, breast, prostate, etc.