Significant progress has been made in our understanding of the molecular lesions responsible for tumor cells to exhibit uncontrolled growth while circumventing normal mechanisms of apoptosis and their ability to migrate and invade normal tissues while evading recognition and destruction by the immune system.

This understanding has enabled the development of therapies specifically targeted to these lesions coupled to innovative treatment regimens to most effectively use these new targeted therapies with precision in selected subpopulations of patients. Innovation at the scientific and clinical levels has been appropriately embraced and supported at the FDA, resulting in regulatory innovation to facilitate and adapt to the Precision Medicine environment.

Starting in the late 1980’s, there was an explosion in our understanding of the molecular lesions that cause and maintain the transformed phenotype. These lesions enable deregulated cell growth, immune evasion, metastatic capability, and escape from apoptosis and senescence. This molecular understanding has resulted in the development of lesion-specific, targeted therapies linked with validated predictive biomarker test to identify tumors with these specific genetic lesions. These targeted therapies have changed the paradigm for oncology drug development and clinical use into a model of Precision Medicine.

Progress continues to be made in the treatment of cancer. In April 2018, the National Cancer Institute released its latest statistics on overall cancer deaths in the U.S. Cancer mortality is dropping at yearly rates of 1.8% for men, 1.4% for women, and 1.4% for children. Better prevention, earlier detection, and knowledge of the causative genetic lesions coupled to targeted therapies all play a part in this progress.


This blog is an edited version of “Innovation in Oncology Drug Development” which was published on 23 November 2019 in the Journal of Oncology. To view the full article, please visit

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ICON's Oncology experts provide analysis including whitepapers, blogs and contributions to media and industry conversations relating to all aspects of oncology in clinical trials.

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