Why real-world evidence has become a regulatory priority
Over the past decade, real-world data and real-world evidence have moved from the periphery of regulatory discussions to the centre of policy debates. Advances in digital health, electronic health records, registries and connected medical devices have created unprecedented opportunities to collect data reflecting actual clinical use rather than controlled trial settings.
FDA’s latest announcement reflects a growing recognition that traditional clinical trials alone cannot fully capture device performance, safety and benefit-risk profiles across diverse patient populations and real-world conditions.
The barrier FDA has now removed
Historically, one of the key limitations to the broader use of real-world evidence in FDA regulatory reviews was the requirement that certain data sources be collected under conditions closely resembling traditional clinical investigations. This constraint reduced the practical usability of many real-world datasets, despite their clinical relevance.
The new FDA policy explicitly removes this barrier, allowing appropriately designed and well-documented real-world data sources to support regulatory submissions without the need to artificially align them with classical trial frameworks.
What changes in practice under the new FDA approach
Under the revised framework, FDA confirms that real-world evidence may be used more broadly to support:
- pre-market submissions for medical devices,
- post-approval safety and performance evaluations,
- label expansions and modifications,
- comparative effectiveness and benefit-risk assessments.
Crucially, the emphasis shifts from the origin of the data to its quality, relevance and fitness for purpose. Methodological rigor, transparency and appropriate statistical analysis become the decisive factors.
Strengthening lifecycle-based evidence generation
FDA’s decision reinforces a lifecycle-based approach to evidence generation, in which pre-market and post-market data are treated as complementary rather than hierarchical. Real-world evidence is positioned as a tool not only for post-market surveillance, but also for informing earlier regulatory decisions.
This approach aligns with FDA’s broader regulatory science agenda and reflects a more adaptive understanding of how medical technologies evolve in real clinical environments.
Implications for medical device manufacturers
For manufacturers, the FDA policy shift creates new strategic opportunities but also new responsibilities. Companies may now:
- leverage registries, electronic health records and device-generated data more effectively,
- reduce reliance on large, traditional clinical trials in certain contexts,
- design evidence strategies that extend seamlessly from pre-market to post-market phases.
At the same time, manufacturers must ensure robust data governance, clear documentation of data provenance and scientifically sound analytical methods.
Implications for IVD manufacturers
For in vitro diagnostic manufacturers, the enhanced role of real-world evidence is particularly relevant in areas such as companion diagnostics, laboratory-developed tests and diagnostics used in decentralised or routine care settings. Real-world evidence may support claims related to clinical performance, clinical utility and population-level impact.
However, FDA emphasises that analytical validity and scientific validity remain non-negotiable foundations, even as greater flexibility is introduced at the clinical evidence level.
A point of reference for global regulators
Although FDA’s decision applies within the U.S. regulatory system, its influence extends globally. Other regulators, including those in the European Union, closely monitor FDA policy developments in the area of regulatory science and evidence generation.
The FDA’s explicit endorsement of real-world evidence may therefore serve as a reference point for ongoing discussions around MDR and IVDR, particularly in relation to post-market surveillance, post-market clinical follow-up and the broader use of real-world data in conformity assessment.
Comparing FDA and EU approaches to real-world evidence
While MDR and IVDR already place strong emphasis on post-market data, the FDA’s approach goes further in explicitly recognising real-world evidence as a potential primary source of regulatory support in certain scenarios. This contrast highlights differing regulatory philosophies but also reveals areas of potential convergence.
Both systems increasingly acknowledge that continuous data generation across the product lifecycle is essential for effective regulation of modern medical technologies.
Strategic considerations for globally active manufacturers
For manufacturers operating in both the U.S. and EU markets, FDA’s policy shift underscores the importance of designing globally coherent evidence strategies. Real-world evidence programmes that meet FDA expectations may, with appropriate adaptation, also strengthen EU post-market surveillance and clinical evaluation activities.
Early alignment of data collection, governance and analysis frameworks can therefore deliver regulatory efficiencies across jurisdictions.
Conclusions
FDA’s removal of a major barrier to the use of real-world evidence marks a significant evolution in regulatory thinking. By prioritising data quality and relevance over rigid methodological formalism, FDA has opened the door to more flexible, lifecycle-oriented evidence generation. For the medical device and IVD sector, this shift not only reshapes U.S. regulatory practice but also contributes to a broader global redefinition of how evidence is generated, assessed and used to protect patients while enabling innovation.