Comparative-effectiveness research (CER) is the current gold standard for improving health care. New products including drugs, medical devices, diagnostics, biomarkers and therapeutics should be proven more effective and safe as compared to standards that are used in a day-to-day clinical practice. International and national Organizations or Agency, such as FDA require rigorous criteria for approval of new diagnostics and therapeutics usually met in large randomized controlled trials. However, such an evaluation does not consider differences in therapy response and adverse effects among patients with same disease who receive the same treatment.
Based on the recent DNA sequencing technology explosion that reveals a widespread genetic variation among patients, a similar explosion over the last few years has been occurred on research for personalized medicine. Aim is to discover those causal mutations characteristic for each patient, which will allow the development of specific individualized therapies. However, there is critique that it is too early for personalized diagnostics and therapeutic approaches and this human genome heterogeneity-based research requires major costs and funding with uncertain effectiveness.
This perspective article discusses strengthens and weaknesses of CER and personalized medicine for improving health care. It is particularly considered costs, funding priorities by agencies and potential harms for the population when very expensive new targeted therapies, for example in cancer treatment, are promoted by industry in the pharmaceutical market with uncertain effectiveness and safety.
(Citation: Gastric & Breast Cancer 2010; 9(3): 95-101)