MASTER KEY Project: Clinical Development for Rare Cancers through a Registry Platform Trial

Abstract

Rare cancers have a challenge in establishing standard therapies compared to major cancers. Critical delays in anticancer drug development, especially for cancers defined as an annual incidence of <6 cases per 100,000 population, have been an issue. To overcome obstacles, MASTER KEY Project was launched in May 2017, which is a multicenter study based in Japan, with two main parts: prospective registry study and multiple clinical trials. Advanced rare cancers, cancers of unknown primary origin, and those with rare tissue subtypes of common cancers are targeted. The registry study accumulates highly reliable consecutive data including patients’ treatment progress and that can be used for future drug development. The multiple trials are conducted simultaneously, targeting either a specific biomarker or a rare tumor type of interest. This project is expected to accelerate development of treatments for rare cancers and show that comprehensive platform trials are an advantageous strategy.