ABSTRACT
Culture in the biomedical research arena plays an enormous role in both the lack of productivity and the focus on common conditions as well. Rare disease drug discovery resides in this difficult morass, and is then only further complicated by small cohorts that make it difficult to characterise the disease and create adequate trial size. Heterogeneity in disease pathophysiology plagues rare conditions, although it affects common ones as well. Regulatory agencies such as United States Food and Drug Administration, Health Canada and the European Medicines Agency examine the safety and efficacy of potential therapies. Even if a validated biomarker is available, it is difficult to get a large enough pool of people for studies to have high enough 'power'. Stratified medicine challenges the traditional drug development paradigm because it creates smaller cohorts and reduces the power of studies. Potential therapies often fail during the clinical trial process. This is part of what adds to the enormous costs of drug development.
