Real-world evidence is derived from curating, standardizing, and analyzing real-world data to obtain high-quality and reliable information from diverse and complex sources.
Evidence generated by traditional clinical research often fails to address key real-world questions of patients, physicians, and health systems regarding the appropriate role of new treatments. These include effectiveness, tolerability, value and heterogeneity of treatment effects.
Bringing a new treatment, from discovery to market, involves significant time and expense. For drug developers of new treatments, broadening research to address real-world questions may introduce additional uncertainty or delay and, in addition, require data not readily available.
Real-world evidence regarding new treatments should address the practical questions of various stakeholders:
For patients and physicians: When is a new treatment preferred over existing alternatives?
For payers: How does the value of a new treatment compare to existing alternatives?
For industry: Where and when is a treatment likely to deliver the greatest value to customers?
For regulators: How can labeling be more informative for patients and clinicians?
Expanding the evidence-generating process to address real-world issues will almost always require more flexible treatment protocols and more heterogeneous clinical populations. However, the 'noise' introduced by that flexibility and heterogeneity could certainly interfere with the detection of primary 'signals' regarding efficacy and safety.