Written by NCI Advisor to Associate Director, Gary J. Kelloff, M.D.


Precision medicine often is called “the right drug for the right patient at the right dose.” This concept is not new—everyday personalized clinical care follows these tenets. What is new and what will enhance precision medicine are the rapid advances being made in our understanding of the molecular basis of disease. These advances are accompanied by the availability of technology that facilitates evaluation of disease at the single cell level, systems for collecting and analyzing the voluminous data required, and recognition by researchers and regulators that new strategies and tools are needed to define the best treatments for patients. For nearly two decades, cancer has been a primary focus for research on the molecular basis of disease, and so in this Special Section of the DIA Global Forum we take a look at the progress being made in applying this research to precision medicine in oncology.

Kelloff and Sigman begin the Section with an overview of the challenges of precision medicine and the resources and strategies that are being applied to meet these challenges in oncology (Strategies to Meet Precision Medicine Challenges in Oncology – Genomics Plus).

Blumenthal and Kazandjian then describe the efforts of the FDA to develop more effective clinical trial strategies and efficient regulatory pathways for molecularly targeted therapies used in precision medicine; they point to recent approvals of these therapies, and they also touch on efforts to incorporate patient-reported outcomes, data from sources other than clinical trials (e.g., tumor registries and electronic health records), and evaluation of complex, multi-variate assays (e.g., net generation sequencing) used for profiling tumors (Precision Medicine: Therapeutic Successes in Oncology).

Gutman and Thomae comment on our current system of diagnostics development in which clinical utility is not primarily addressed by test developers, and discuss the special case of companion diagnostics wherein there is an opportunity to obtain more transparent, standardized results that would provide better data on value and outcomes (The Eye of the Tortoise: Companion Diagnostics and the Path to Market).

Pacanowski and Leptak continue the discussion on the FDA regulatory framework for precision medicine. Particularly, they describe FDA’s collaborative approach in developing the framework and resources and guidances available from FDA (The Evolving Regulatory Framework for Precision Drugs and Biologics).

Sigal then emphasizes the important role that patient experience has in the development of effective treatments and describes how this experience can be captured for use in regulatory evaluations (Patient Needs Drive Medicine).

Immunotherapy has become the most exciting and promising new treatment strategy in oncology, and so in the last article in the Section, Kaufman and Rubin describe recent research on factors governing response to immunotherapy and on the possibility of precision medicine strategies integrating molecular profile and immunotype of a patient’s cancer (Precision Medicine in the Era of Cancer Immunotherapy).


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