The Growing Role of Real-World Evidence in Regulatory Decisions
For years, clinical trials have been the cornerstone of regulatory approval. But as medicine becomes more complex and data streams more diverse, regulators are looking beyond the walls of traditional studies. Enter real-world evidence (RWE)—data-driven insights drawn from electronic health records (EHRs), patient registries, claims databases, and digital health tools. RWE is increasingly shaping not only product approvals but also post-market surveillance, ensuring therapies and devices are safe and effective in real-world use.
How RWE is Transforming Approvals & Post-Market Surveillance
Unlike controlled clinical trials, which often involve narrow patient populations and standardized protocols, RWE reflects everyday use. By supplementing trial data, RWE allows regulators to better understand how diverse populations respond to therapies and devices, addressing key questions about safety, efficacy, and long-term outcomes.
In approvals: RWE helps fill gaps left by traditional trials, providing evidence from broader, more representative patient groups.
In surveillance: RWE enables regulators to track product performance continuously, identifying safety issues earlier and more precisely.
For example, both the FDA and European Commission have integrated RWE into frameworks for medical devices, drugs, and combination products, recognizing its role in accelerating access to innovation while safeguarding patient safety.
A Word of Caution: The Quality Bar Is High
While the FDA encourages the use of RWE, not all real-world data qualifies as real-world evidence. Regulators emphasize that only high-quality, reliable, and well-controlled RWE can support regulatory decision-making.
In NMCG’s experience, this is one of the biggest misconceptions sponsors face. Companies often assume that because a product has been on the market overseas for years with no issues, that data automatically qualifies as RWE. The FDA does not view that as quality evidence.
According to the FDA’s RWE Framework, to be considered regulatory-grade RWE, data must be:
Fit for purpose—collected systematically and analyzed using scientifically sound methods.
Traceable and verifiable—with documentation that meets Good Clinical Practice (GCP) standards.
Relevant to U.S. use and patient populations, when supporting a U.S. submission.
In many cases, RWE is best used to justify more efficient and targeted pivotal studies, rather than to replace them outright—especially for moderate- to high-risk devices.
Strengthening Post-Market Surveillance with RWE
Post-market surveillance has always been essential to regulatory oversight. What’s changing is the integration of big data, AI, and wearable technology to detect emerging risks in real time.
By drawing on EHRs, patient-reported outcomes, and connected devices, regulators and manufacturers can:
Detect safety signals earlier
Monitor product performance over longer time horizons
Ensure compliance with evolving standards
Maintain public trust through transparency and responsiveness
At NMCG, we see clients preparing for this shift by incorporating regulatory-ready data practices from the start—ensuring evidence collected in trials can flow seamlessly into post-market use.
Global Adverse Event Reporting Systems
Adverse event (AE) reporting remains the backbone of global pharmacovigilance and device monitoring. Systems such as:
FDA’s FAERS (U.S.) – A database for adverse event reports submitted by clinicians, consumers, and manufacturers.
WHO’s VigiBase – A global repository supported by 150+ countries, offering tools like VigiFlow for submissions and VigiLyze for signal detection.
These systems:
Collect: Reports from healthcare professionals, patients, and companies.
Analyze: To detect safety signals and trends.
Act: Trigger regulatory decisions like label changes, warnings, or recalls.
However, challenges remain—including data quality, timeliness of reporting, and system interoperability across regions. Integrating RWE into these systems may help overcome these gaps, improving signal detection and harmonization worldwide.
Why It Matters for Sponsors
For innovators, the growing role of RWE means adapting early:
Design protocols that anticipate both premarket and post-market data needs.
Prioritize data quality—fit-for-purpose, verifiable, and aligned with FDA expectations.
Build robust quality systems that meet ISO 13485 and global PMS standards.
Leverage experienced regulatory partners to ensure RWE supports—not hinders—your submission strategy.
Why Partner with NMCG?
Our Regulatory and Clinical services help clients design studies and data strategies that are future-proof—bridging the gap between trial evidence and real-world performance while meeting FDA's high standards for data reliability and validity.
