Real world data (RWD) and Real world evidence (RWE) in Drug Development

The 21st Century Cures Act (Cures Act), signed into law on December 13, 2016, is designed to accelerate medical product development and bring new innovations and advances faster and more efficiently to the patients who need them. Following the passing of the Cures Act, the Food and Drug Administration (FDA) has created a framework for evaluating the potential use of real-world evidence (RWE) to help support the approval of a new indication for a drug already approved or to help support or satisfy drug postapproval study requirements.

In December, 2018, FDA issued "Framework for FDA’s Real-World Evidence Program" and FDA's CDER and CBER divisions (now also including the oncology center of excellence) created the RWE program. A series of guidance documents were released. 

DEFINITION of RWD and RWE:

FDA GUIDANCE DOCUMENTS on RWD and RWE (as of November 2024):

Topic	Title	Category	Current Status
EHRs and claims data	Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products	Data considerations	Final, July 2024
Registry data	Real-World Data Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products	Data considerations	Final, December 2023
Data standards	Data Standards for Drug and Biological Product Submissions Containing Real-World Data	Data submission	Final, December 2023
Regulatory considerations	Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products	Applicability of regulations	Final , August  2023
Submitting RWE	Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drug and Biological Products	Procedural	Final, September 2022
Externally controlled trials	Considerations for Design and Conduct of Externally Controlled Trials for Drug and Biological Products	Design considerations	Draft, February 2023
Non-interventional studies	Considerations Regarding Non-Interventional Studies for Drug and Biological Products	Design considerations	Draft, March 2024
RCTs in clinical practice settings	Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice	Design considerations	Draft, September 2024

WEBINARS for RWD/RWE:

FDA officials have given various webinars to explain these RWD/RWE guidance documents and encourage the sponsors to apply the RWE to the drug approval process. A non-profit organization, the Reagan-Udall Foundation for the FDA, in collaboration with the Food and Drug Administration (FDA), hosted a series of free, public webinars to discuss FDA-issued guidance in the RWD/RWE. 

Title	Webinar Series	Date
Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products	https://reaganudall.org/news-and-events/events/public-webinar-series-fda-issued-guidance-real-world-evidence	November 4, 2021
Data Standards for Drug and Biological Product Submissions Containing Real-World Data	https://reaganudall.org/news-and-events/events/real-world-data-webinar-series-data-standards	December 3, 2021
Real-World Data Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products	https://reaganudall.org/news-and-events/events/real-world-data-webinar-series-registries	January 28, 2022
Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products	https://reaganudall.org/news-and-events/events/real-world-data-webinar-series-considerations-use-rwd-and-rwe	February 11, 2022
Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drug and Biological Products	No webinar was conducted
Considerations for Design and Conduct of Externally Controlled Trials for Drug and Biological Products	https://www.youtube.com/watch?v=5rfInDy7osw&t=1s	April 13, 2023
Considerations Regarding Non-Interventional Studies for Drug and Biological Products	https://reaganudall.org/news-and-events/events/real-world-evidence-webinar-series-considerations-regarding-non	May 30, 2024
Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice	https://reaganudall.org/news-and-events/events/real-world-evidence-webinar-series-integrating-randomized-controlled-trials https://youtu.be/VRaQyOvn3AM?si=YrM9pY6JhL3LBr_o	November 22, 2024

Duke Margolis Center for Health Policy, in collaboration with the FDA, also conducted a series of free, public webinars to discuss the application of RWD/RWE: 

Webinar Title/Link	Date
Optimizing the Use of Real-World Evidence in Regulatory Decision-Making for Drugs and Biological Products – Looking Forward	December 12, 2024
2024 State of Real-World Evidence Policy	July 25, 2024
The State of Real-World Evidence Policy 2023	September 28, 2023
Understanding the Use of Negative Controls to Assess the Validity of Non-Interventional Studies of Treatment Using Real-World Evidence	March 8, 2023
Workshop on Draft Guidance on Real-World Data: Electronic Health Records/Medical Claims Data and Data Standards	February 27, 2023
The State of Real-World Evidence Policy	May 12, 2022
An Introduction to Real-World Data & Real-World Evidence: A Virtual Training Series for the Patient Community	March 12, 2021

SUMMARY:

RWD / RWE play an increasingly vital role in drug development by complementing traditional clinical trial data. Derived from sources such as electronic health records, insurance claims, registries, and patient-reported outcomes, RWD provides insights into how drugs perform in diverse, routine care settings. RWE, generated by analyzing RWD, helps assess the safety, efficacy, and value of treatments in real-world populations, addressing gaps that controlled clinical trials may leave. These insights are particularly valuable in identifying long-term outcomes, supporting regulatory decisions, designing pragmatic trials and comparative effectiveness researches, and informing post-market safety surveillance. Regulatory agencies like the FDA and EMA are encouraging the integration of RWE to enhance decision-making, optimize study designs, and support label expansions or accelerated approvals.