Novel medicines that target the underlying causes of the disease are improving the outlook for many patients. But behind every medicine that makes it to patients there are many investigational medicines that fail. The biopharmaceutical pipeline includes many of these so-called “failures” which should more appropriately be considered setbacks. The nature of conducting research in areas of high scientific complexity and regulatory uncertainty is that failure is inevitable. The knowledge gained help inform future research and development projects, including new therapeutic strategies and potential treatment combinations.