Let me be direct: if your organization still treats real-world evidence as a "nice to have" supplement to randomized controlled trials, you are already behind. The FDA has moved. The question is whether you have moved with them.
I have spent over a decade working at the intersection of healthcare data and evidence generation, and I have never seen a regulatory shift this decisive happen this quietly. While most of the industry was debating whether RWE would ever "count," the FDA was building an entire infrastructure to make it not just acceptable but expected. The numbers tell the story clearly -- since 2016, more than 35 drugs and biologics and over 250 medical devices have incorporated real-world evidence in their FDA submissions. This is not a pilot program. This is the new normal.
What Actually Changed
Three regulatory developments in the past year should have every evidence leader rethinking their strategy. If you missed any of them, pay attention now.
First, the December 2025 privacy breakthrough. The FDA eliminated the longstanding requirement that real-world evidence submissions include identifiable individual patient data. Read that again. For years, one of the biggest barriers to scaling RWE was the tension between regulatory rigor and patient privacy. Researchers had to navigate complex de-identification protocols, data use agreements, and institutional review board concerns -- all because the FDA wanted to see identifiable records. That barrier is gone. This is a massive win for both patient privacy and the practical scalability of RWE programs. Organizations that were holding back on certain data sources because of re-identification risk now have far more room to operate.
Second, the 2026 Regulatory Science Framework. The FDA is not just accepting RWE -- they are actively investing in making it better. Their 2026 framework is soliciting proposals specifically aimed at strengthening real-world data methods. This is the agency saying, in plain terms, "We want this to work, and we want the science to be rigorous enough that we can rely on it for consequential decisions." When the regulator is funding methodology improvements for your evidence type, that is as strong a signal as you will ever get.
Third, the technology infrastructure play. The FDA is exploring the HL7 FHIR standard for receiving clinical study data from real-world data sources. This is a technical detail that has enormous strategic implications. FHIR is the interoperability standard that most health systems are already adopting for clinical data exchange. If the FDA builds intake pipelines around FHIR, it creates a direct line from electronic health records to regulatory submissions. The friction cost of using RWD drops dramatically. Think about what that means for the speed and cost of evidence generation.
Taken together, these three shifts represent something more than incremental progress. They represent a regulatory body that has made a structural commitment to real-world evidence as a primary input to its decision-making.
Where RWE Is Winning
The abstract policy shifts matter, but what convinced me that RWE has permanently crossed the threshold is the label expansion data.
Between 2022 and 2024, somewhere between 23% and 28% of FDA label expansions utilized real-world evidence. Not supplemented by. Not footnoted with. Utilized. That is roughly one in four label changes relying on evidence generated outside the traditional clinical trial apparatus.
The distribution is telling. Oncology leads at 43.6% of RWE-supported label changes, which makes intuitive sense -- oncology has always been the frontier for evidence innovation because the urgency of the disease and the complexity of treatment pathways demand it. But this is not an oncology-only story. The trend lines across therapeutic areas are all moving in the same direction.
Here is the detail that should reshape how you think about your evidence strategy: 78.2% of RWE-supported label changes were for indication expansion. Not safety updates. Not post-market commitments. Indication expansion -- the highest-value regulatory outcome a brand team can pursue. Real-world evidence is not being used for low-stakes housekeeping. It is being used for the decisions that drive revenue and patient access.
The study designs powering these submissions are not exotic. Most are retrospective cohort studies using electronic health record data. This is not cutting-edge methodology requiring specialized infrastructure. This is bread-and-butter epidemiology applied with discipline and rigor to data that already exists in health systems. The barrier to entry is not technical sophistication. It is organizational commitment to doing it well.
The Data Quality Imperative
And this is where I need to push back on a narrative I see constantly in this space: the idea that more data is automatically better.
It is not. I have reviewed enough RWE submissions and study protocols to tell you with confidence that the single biggest differentiator between evidence that moves the needle and evidence that gets filed away is data quality. Not data volume. Not data novelty. Quality.
When the FDA reviews an RWE submission, they are asking a specific set of questions. Is the data source fit for purpose? Are the variable definitions clinically meaningful? Has confounding been addressed with appropriate rigor? Is the study design aligned with the causal question being asked? No amount of data will compensate for a poorly defined exposure variable or a confounding structure that the analysis plan did not account for.
This is why I keep coming back to the same principle in my work and in my RWEdnesdays content: quality over quantity, every single time. A well-designed retrospective cohort study using a single, deeply characterized EHR data source will outperform a sloppy analysis of a billion patient records from a claims database where you cannot validate the phenotype definitions.
The FDA's investment in the 2026 Regulatory Science Framework reinforces this point. They are not asking for proposals to make datasets bigger. They are asking for proposals to make methods stronger. Fit-for-purpose assessments. Bias quantification frameworks. Sensitivity analyses that stress-test assumptions. The agency knows that the future of RWE depends on methodological discipline, not data accumulation.
The rare disease space illustrates this perfectly. Half of the novel drugs the FDA approved in 2024 carried orphan drug designation, and the FDA launched its Rare Disease Innovation Hub in July 2024 specifically to support development in this area. In rare diseases, large datasets often do not exist. The path to evidence is through deep, careful characterization of small populations -- exactly the scenario where data quality is everything and data quantity is a constraint you work around, not a feature you rely on.
What This Means for Your Organization
If you are a VP or Director at a pharma company reading this, here is my honest assessment of where the opportunity sits.
The economic case is now undeniable. The RWE market has grown from $2.3 billion in 2023 to a projected $4.5 billion by 2029. Deloitte's research suggests that integrating real-world data with AI-driven analytics could reduce drug development costs by up to 60%. Top-10 pharma companies are looking at $500 million to $1 billion in annual savings. These are not speculative numbers -- they reflect the compounding value of faster evidence generation, more efficient trial designs, and regulatory pathways that do not require you to run a 5,000-patient RCT for every label expansion.
But the savings only materialize if you invest in the right capabilities. I see too many organizations treat RWE as a procurement problem -- buy the biggest dataset, hand it to a CRO, check the box. That approach will produce evidence that does not hold up to regulatory scrutiny and will not give you the competitive advantage you need.
Here is what I would prioritize:
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Build internal expertise in RWD quality assessment. You need people who can evaluate whether a data source is fit for the specific regulatory question you are trying to answer. This is a specialized skill that combines clinical knowledge, epidemiological training, and regulatory awareness.
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Invest in your data infrastructure now. The FDA's move toward FHIR-based data intake is a signal. Organizations that build interoperable data pipelines today will have a structural advantage when those pathways formalize. If your real-world data strategy still depends on flat files and manual curation, you are building on a foundation that will not scale.
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Stop treating RWE and RCTs as competitors. The most sophisticated evidence strategies I see use RWE to inform trial design, supplement control arms, support label expansions after initial approval, and monitor long-term safety. It is not RWE or RCTs. It is an integrated evidence ecosystem where each approach plays to its strengths.
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Get your regulatory affairs team fluent in RWE now. The December 2025 privacy policy change and the 2026 framework are just the beginning. Regulatory expectations around RWE are going to continue evolving rapidly. If your reg affairs team still views RWE with skepticism or treats it as someone else's problem, you will miss windows of opportunity that your competitors will not.
The Bottom Line
I have built my career on the conviction that better evidence leads to better healthcare decisions. What the FDA has done over the past two years is validate that conviction at the highest regulatory level. They are not just tolerating real-world evidence -- they are building the infrastructure to make it a cornerstone of how drugs and devices are evaluated.
But here is the part that keeps me up at night: the same accessibility that makes RWE powerful also makes it easy to do poorly. The barrier to generating real-world evidence has dropped. The barrier to generating good real-world evidence has not. The organizations that win in this environment will be the ones that understand the difference -- the ones that refuse to sacrifice rigor for speed, that invest in methodology as seriously as they invest in data access, and that treat every RWE submission as an opportunity to demonstrate that this evidence type deserves the trust the FDA is placing in it.
The regulatory door is open. The question is whether you are going to walk through it with evidence that can stand on its own -- or evidence that undermines the case for everyone who comes after you. I know which side of that line I intend to be on.