Generate Real-World Data (RWD) and Real-World Evidence (RWE) for Regulatory Purposes

Real-world data (RWD) and real-world evidence (RWE) have been hot topics in the drug development field and in the statistical field. With the new technologies, electronic health records, and big data, it is no surprise that RWD and RWE are much discussed to be a new way to revolutionize the clinical trial design and the regulations.

FDA has its dedicated webpage for 'Real-World Evidence' and issued several guidelines for using real-world evidence to support the regulatory approvals. 

What is the Definition of RWD and RWE?

Real-world data (RWD) are the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. RWD can come from a number of sources, for example:

• Electronic health records (EHRs)
• Claims and billing activities
• Product and disease registries
• Patient-generated data including in home-use settings
• Data gathered from other sources that can inform on health status, such as mobile devices

Real-world evidence (RWE) is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD. RWE can be generated by different study designs or analyses, including but not limited to, randomized trials, including large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective).

Last week, Duke Margolis Center for Health Policy organized a symposium titled "Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes". All presentation slides and the video recordings are available for free.

Day 1 focused on the study design using RWD and RWE for efficacy measure (presentation slides, presentation video)

Day 2 focused on safety monitoring using RWD and RWE (presentation slides, presentation video)

This is not the first Duke Margolis Center for Health Policy organizes the event for this topic. Here are some previous events.

Second Annual Duke-Margolis Conference on Real-World Data and Evidence

Enhancing the Application of Real-World Evidence In Regulatory Decision-Making DAY 1

Public Workshop: A Framework for Regulatory Use of Real-World Evidence

Additional Reading: 

• Real-World Evidence — What Is It and What Can It Tell Us? NEJM 2018
• FDA guidance (2018) "Use of Real-World Evidence to Support Regulatory Decision-Makingfor Medical Devices"

• FDA Discusses RWD, RWE With Industry, Academia
• Good practices for real‐world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR‐ISPE Special Task Force on real‐world evidence in health care decision making Pharmacoepidemiology and Drug Safety and Value in Health
• Real-World Evidence and Real-World Data for Evaluating Drug Safety and Effectiveness
• Gilead and Novartis Seek to Expand What FDA Considers as Real-World Data and Real-World Evidence