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Authors
Advisor(s)
Abstract(s)
Cancer diagnosis and therapeutics have been traditionally based on pathologic classification at the organ of origin. The availability of an unprecedented amount of clinical and biologic data provides a unique window of opportunity for the development of new drugs. What was once treated as a homogeneous disease with a one-size-fits-all approach was shown to be a rather heterogeneous condition, with multiple targetable mutations that can vary during the course of the disease. Clinical trial designs have had to adapt to the exponential growth of targetable mechanisms and new agents, with ensuing challenges that are closer to those experienced with rare diseases and orphan medicines. To face these problems, precision/enrichment and other novel trial designs have been developed, and the concept of histology-agnostic targeted therapeutic agents has emerged. Patients are selected for a specific agent based on specific genomic or molecular alterations, with the same compound used to potentially treat a multiplicity of cancers, granted that the actionable driver alteration is present. There are currently approved drugs for such indications, but this approach has raised issues on multiple levels. This review aims to address the challenges of this new concept and provide insights into possible solutions and frameworks on how to tackle them.
Description
© The Author(s) 2022. Open Access This article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/.
Keywords
Early drug development Enrichment design New trial design Precision medicine Tissue-agnostic drug
Pedagogical Context
Citation
Adv Ther. 2022 Nov 23
Publisher
Springer Nature