In our data-rich world, key medical decisions, ranging from a regulator’s decision to curtail a drug to patient-specific treatment choices, require optimal consideration of myriad inputs. Statistical/epidemiological methods that can harness real-world medical data in useful ways do exist, but much work remains to achieve the full potential of a truly data-driven user-centric medical environment. I will lay out some of the key challenges before us and describe recent progress in the specific area of drug safety.