Drug Development in Tissue-Agnostic Indications.

ABSTRACT: A better understanding of cancer biology has led to the development of targeted therapies specifically designed to modulate an altered molecular pathway in the cancer cells or their microenvironment. Despite the identification of molecular targets across cancer types, most of targeted therapies were developed per cancer type. In this ancestral paradigm, randomization was the gold-standard approach for market access. Randomization of large patient populations was feasible for drugs developed in common cancer types but more challenging in rare cancer types. The traditional paradigm of drug development in oncology was further challenged by the ever-expanding molecular segmentation of cancer with ever-smaller subgroups of patients who might benefit from specific targeted therapies or immunothe

SUBMITTER: du Rusquec P 

PROVIDER: S-EPMC8199632 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature