The double-blind, placebo-controlled clinical study has been and continues to be the gold standard in pharmaceutical research. While that remains fundamental to evaluating the safety and efficacy of any drug, real-world data analyses can give us insight to what happens when the medicine is used in the “real world” – where conditions and the variables in each patient’s life may come into play. No matter how thorough our clinical studies are, there is always more to learn once a medicine goes into that real-world use. As Dr. Tom Frieden, the former director of the U.S. Centers for Disease Control and Prevention, recently wrote for the New England Journal of Medicine, randomized controlled trials can establish a drug’s efficacy, while data from real-world observations are useful to refine dosages and identify side effects, especially rare ones.
In the past, individual clinicians would use their own real-world experience to inform their decisions. Now, electronic health records in hospitals and clinical offices make it possible for health care providers and their patients to contribute de-identified data seamlessly and for researchers to access aggregated data easily. That, plus the development of technology that can process enormous amounts of data, lets us study the real-world experience of large numbers of people. We can potentially spot emerging trends in clinical practice or pinpoint the characteristics of patients that may optimize outcomes.