On Your Marks

Technological advances, treatments targeting chronic diseases, chronic adjuvant or preventative indications, individualised medicine, and mounting development costs have put biomarkers at the forefront of development programmes of both small molecule drugs and biopharmaceuticals. The increased importance has also expanded the meaning of biomarker from any measurable characteristic that predicts or indicates a patient’s normal or disease pathogenesis or response to therapy, to encompass screening assays, the ‘omics’ (genomics, proteomics and metabonomics), specific enzyme and protein measurements, and imaging – not only for disease progression and efficacy but also as markers for adverse events.

While few of these biomarkers qualify as surrogate markers of clinical efficacy for regulatory approval purposes, biomarkers were identified by the US Food and Drug Administration (FDA) as one of the cornerstones of innovation in drug development in the 2004 Critical Path White Paper. Even though the outcome from this white paper has yet to materialise, there is no denying the pivotal and increasing role of biomarkers in the development of pharmaceuticals (1).

PARTNERING FOR BIOMARKER DISCOVERY AND ACCESS TO TECHNOLOGY

The breadth of methodologies, cost associated with the instrumentation, specialised algorithms and informatics requirements, and intellectual property rights have led to the emergence of highly specialised companies. In particular, highthroughput screening with large volumes of interrelated data requires statistical models and pattern recognition that challenge traditional approaches to assessing biological significance. Genomics, proteomics, RNA profiling and gene function analysis have been used in drug discovery programmes to identify novel targets and develop disease models (2), and rapidly evolving cellular imaging technology is being applied to capture effects on complex biological systems (3).