From heat maps to clinical tests: Why so few regulatory approved molecular profiles?
Nicholas C. Dracopoli · Clinical Cancer Research · 2010
Abstract Currently, only about 10% of oncology compounds entering Phase I clinical trials make it all the way to regulatory approval. The main goals of biomarker research in the pharmaceutical industry are to increase efficiency of the drug development process by improving understanding of the mechanism of action, deeper exploration of PK-PD interactions and predicting response to novel therapies in clinical development. The development of novel biomarkers by analysis of multidimensional clinical and biological data has enormous potential to impact drug development and improve patient outcomes. However, no complex molecular or protein profiles have been approved by the FDA to drive therapeutic use of any drug. Predictive markers are described in the labels for only a minority of the oncology drugs approved in the US since Herceptin in 1998. These markers all measure the status of the drug target or pathway and do not involve complex molecular or protein profiles. This presentation will discuss why this is the case and show how the nature of the clinical trial process, the limited numbers of patients enrolled in clinical trials, and the lack of long-term outcome data have severely limited the impact of molecular profiling in the drug approval process and ultimately clinical practice.