Real-World Evidence · Regulatory
FDA and Real-World Evidence
The FDA has built a formal framework for using real-world evidence in regulatory decisions, reshaping how approved treatments are studied and expanded.
The regulatory science behind it is a central thread at RWE Connect 2026 in Boston.
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The framework
The FDA Real-World Evidence Framework
Under the 21st Century Cures Act of 2016, the FDA was directed to build a framework for evaluating whether real-world evidence can help support regulatory decisions, such as new indications for approved drugs and post-approval study requirements.
The result is a growing body of guidance on when and how real-world data can be used to generate evidence the agency will accept. It draws on the wider world of real-world evidence and real-world data.
The key distinction
RWD vs RWE, in FDA Terms
The FDA draws a precise line between the data and the evidence. Getting the distinction right is the starting point for any regulatory use.
Real-World Data (RWD)
- The raw data itself
- EHRs, claims, registries, wearables
- Collected outside clinical trials
- The information
Real-World Evidence (RWE)
- The clinical evidence derived from data
- About a treatment’s use, benefits or risks
- Produced by analyzing RWD
- The conclusions
The use cases
How the FDA Uses Real-World Evidence
Real-world evidence is not a replacement for trials, but it fills gaps trials cannot. These are the main regulatory uses.
The bar to clear
The FDA’s Evidentiary Standards
For real-world evidence to inform a regulatory decision, it has to meet a demanding standard. These are the principles that define regulatory-grade RWE.
How it works
Real-World Evidence in the Regulatory Journey
A regulatory RWE study follows a disciplined path, with early FDA engagement at its heart.
Many of these studies rely on external control arms and the wider RWE methods toolkit.
The direction of travel
The State of FDA RWE in 2026
Regulatory use of real-world evidence is maturing fast, with clearer standards and wider acceptance. These are the shifts on the agenda at RWE Connect 2026.
Who is in the room
Who Attends RWE Connect 2026
A senior, focused audience of the people who generate regulatory-grade real-world evidence.
Seniority is confirmed: 150+ attendees, around 60% at Director or VP level and above. The functional mix shown is indicative; confirmed registration splits are shared with sponsors from the live delegate list.
Explore
Explore Related Topics
Regulatory RWE builds on the wider world of real-world data and methods.
Sponsor RWE Connect 2026
Meet the Regulatory RWE Community in Boston
The teams generating regulatory-grade real-world evidence gather at RWE Connect 2026: 150+ attendees, around 60% at Director or VP level and above.
RWE Connect 2026
Get the RWE Pipeline Report 2026
See the evidence trends reshaping how regulators use real-world data, then meet the community in Boston.
- How the FDA framework is evolving
- What counts as regulatory-grade RWE
- Where regulatory acceptance is heading
Free. Instant download. No obligation.
FDA and Real-World Evidence: Common Questions
What is the FDA’s real-world evidence framework?
The FDA’s real-world evidence framework is the set of principles and guidance, mandated by the 21st Century Cures Act, for evaluating whether real-world evidence can support regulatory decisions on drugs and biologics.
What is the difference between RWD and RWE in FDA terms?
Real-world data (RWD) is the raw data collected outside trials, such as EHRs, claims and registries. Real-world evidence (RWE) is the clinical evidence about a treatment’s use, benefits or risks derived from analyzing that data.
What is the 21st Century Cures Act?
A 2016 US law that, among other things, directed the FDA to create a framework for using real-world evidence to support new indications and post-approval study requirements for approved drugs.
How does the FDA use real-world evidence?
To support label expansions and new indications, monitor post-market safety, contextualize single-arm trials with external controls, and inform regulatory decisions.
Can real-world evidence support drug approval?
Yes, in appropriate cases. RWE can support new indications and label changes for approved products when the data and study design are fit for the regulatory question.
What is regulatory-grade real-world evidence?
RWE built to the standards the FDA expects: relevant and reliable data, fit-for-purpose sources, rigorous pre-specified study design, clear data provenance and full transparency.
What is fit-for-purpose data?
Data that is adequate to answer a specific regulatory question, judged on its relevance and reliability for that particular use, rather than in general.
What is the FDA Sentinel system?
Sentinel is the FDA’s national system for monitoring the safety of approved medical products using real-world data from a large network of healthcare sources.
Are external control arms accepted by the FDA?
In appropriate settings, yes. External control arms built from real-world or historical data can support single-arm trials, most established in oncology and rare disease.
How does real-world evidence relate to drug safety?
Real-world evidence underpins post-market safety surveillance, detecting and characterizing adverse events across large populations after a product is approved.
What is RWE Connect 2026?
RWE Connect 2026 is a two-day real-world evidence summit in Boston bringing together the RWE, HEOR and regulatory leaders who generate and apply real-world evidence, with 40+ speakers and 150+ attendees.
When and where is RWE Connect 2026?
14 to 15 October 2026 at the Hampton Inn by Hilton, Boston Seaport District, Boston, MA.
