Clinical trial design in rare mutation tumors

Abstract This review examines the clinical trial designs in rare mutation tumors, defined as mutations occurring in ≤ 5% of specific cancer types. It discusses molecular characteristics, the impact of histological types, and the complexities of trial conduct due to tumor heterogeneity and limited patient numbers. The article explores accelerated approval pathways, adaptive trial methods like basket and telescope designs, and the use of surrogate endpoints. It underscores the significance of international collaboration and technological advancements in advancing research for these tumors. The review concludes that rare mutation tumor presents a novel prospective for oncological researches and facilitate the precision treatment of tumors.