Frequently Asked Questions

Real-World Data/Real-World Evidence

Real-word data (RWD) and real-world evidence (RWE) are considered by the FDA (as part of the 21st Century Cures Act) to be highly credible and critical sources of information to help guide clinical decision-making for life-saving therapeutic interventions. This data represents the missing link between the prescribed clinical intervention and the patient’s real-time, unique, physiological response.

Clinical research trials are the gold standard for many conditions, however, especially in the case of new or rare diagnoses, we don’t have the ability to engage in a controlled trial. Creating a control group in a traditional clinical setting would fail to provide those patients with the appropriate standard of medical care. It’s unreasonable to expect patients who present to the hospital with life-threatening symptoms to participate in a study that very well may include them receiving a placebo rather than a life-saving treatment.

In this sense, real-world data can provide insights that even a clinical trial cannot; the information garnered represents outcomes of various treatments prescribed by qualified medical professionals using their best clinical judgment to treat their patients in a real-world setting.  These unique findings support the reason Congress mandated that the FDA use RWD in making regulatory decisions.