Science

July 29, 2016

By Spencer Phillips Hey and Aaron S. Kesselheim

The goal of precision medicine (PM) is to “ensure that the right treatment is delivered to the right patient at the right time” (1). Predictive biomarker diagnostics are critical to this effort. Yet despite substantial promise, PM has been plagued with problems (2). Many commercially available biomarker diagnostics have not been adequately validated (3); the scientific literature is flooded with low-quality and unreliable reports (2); and even ostensibly successful PMs, such as trastuzumab (Herceptin) chemotherapy in HER2-expressing breast cancer, have been characterized by uncertainty about how to use and interpret diagnostic test results (4). These disappointing features of PM research can be explained by three obstacles inherent in the science: (i) Biological theories play a central role in the testing methodology; therefore, pivotal trials of PMs cannot be agnostic about underlying mechanisms. (ii) Interventions are complex with many components and degrees of uncertainty that need to be resolved before clinical use. (iii) No single stakeholder controls the biomarkers or coordinates the research program. Although some new regulatory (5) and evidence synthesis (6) efforts are designed to address these problems, we believe that meaningful progress in PM requires new mechanisms of scientific oversight.

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