Beyond the Trial: How Real-World Evidence Is Reshaping Access, Outcomes, and Digital Health 

For decades, clinical trials have served as the gold standard for determining whether a treatment is safe and effective for use by the public. Yet leaders in medicine, market access, and digital health are increasingly confronting the same question: Can controlled trials fully capture how patients behave, respond, and improve in the real world? 

The answer emerging across multiple therapeutic areas is often no. 

Clinical trials remain essential. They provide the rigor, standardization, and oversight necessary for initial evaluation of safety and efficacy. But they are also conducted with structured designs that rarely reflect the complexities of the real world, including the true diversity of the patient population, the challenges with ensuring adherence, and the heterogeneous treatment patterns of providers. These factors can and do dramatically influence whether a treatment works once it leaves the controlled trial setting. In other words, clinical trials are necessary but not sufficient.

As healthcare moves toward more personalized, digital, and at-home care models, real-world evidence (RWE) is becoming a critical complement to traditional trials for assessing the true benefit of new treatments. 

Why RWE Matters Now 

The FDA defines real-world data as health information routinely collected from sources such as registries, electronic health records, or digital health technologies. RWE, in turn, is the insights generated from analyzing these datasets. In guidance published over the last several years, the FDA has repeatedly emphasized the growing role of RWE in supporting regulatory decisions, including label expansion applications and post-market evaluation efforts. 

RWE is especially powerful for pediatric populations, where adherence, convenience, and family lifestyle can drastically influence outcomes in the real world. 

The Case for RWE in Digital Therapeutics and Pediatric Care 

In healthcare broadly, real-world outcomes often diverge from clinical trial results, which is a dynamic known as the efficacy-effectiveness gap. Trials take place in controlled environments with structured follow-up and closely supported use conditions. But once a therapy enters everyday life, factors such as patient behavior, variable adherence, differing care routines, and environmental influences can meaningfully impact how well an intervention performs. For digital health solutions in particular, real-world usage patterns, daily workflows, and comfort with technology all shape effectiveness in ways that trials alone cannot fully capture.

RWE can reveal these patterns at scale, showing not just whether a therapy can work under ideal conditions, but whether it does work in everyday life. Healthcare practitioners want to make sure the treatments they’re prescribing are having a positive impact on their patients, so paying close attention to these patterns is of the utmost importance. 

As the Co-Founder and CEO of a U.S.-based digital therapeutic company that has developed a modern treatment for pediatric amblyopia (commonly known as lazy eye), I have personal experience with the growing value of RWE. Our company’s initial FDA clearance for children aged 4 to 7 was supported by a Phase 3 randomized controlled trial. What happened next highlights the emerging role of RWE in regulatory approvals for new treatments. 

To understand how the therapy performed in real life, we built a national registry tracking real-world treatment outcomes. To date, the registry has captured data from more than 500 patients, across 45 prescribers and 14 practices. When analyzed, the registry results showed that real-world outcomes with the therapy were right in line with clinical trial performance, demonstrating that unlike other therapies, there was no efficacy-effectiveness gap to worry about in this case 

In 2025, the FDA expanded the age range for our company to include 8- to 12-year-olds based primarily on this RWE, which showed substantial equivalence in efficacy between younger and older children. As far as we are aware, this marked the first instance of a label expansion clearance being granted primarily on the basis of RWE  in ophthalmology. For families, this important development meant broader access, fewer barriers to care, and potentially life-changing intervention during a critical window in children’s development. 

How Real-World Data Strengthens Innovation and Access 

The value of RWE extends beyond FDA regulatory decisions. In many cases, it can accelerate payer coverage and help medical directors better quantify the value proposition of new products. Several healthcare analysts have highlighted this trend, noting that RWE has become one of the most influential factors in payer decision-making for digital health solutions. 

A strong RWE foundation helps answer questions that clinical trials often cannot, including: 

• How does treatment adherence vary across populations? 

• What do the longer-term outcomes look like?
• Are the improvements durable after patients stop treatment?
• Are there specific subgroups who benefit more (or less)? 
• How does the therapy integrate into existing family, school, or work routines? 

The answers to these questions are key to outcome success. 

The Future: Blending Trials With Real-World Learning 

The long-term trajectory of evidence generation is clear: clinical trials will remain essential, but they will increasingly be paired with real-world evidence. 

This shift is aligned with the FDA’s broader modernization efforts, as well as a global push toward value-based care. A 2024 analysis in Eye underscored this direction, noting that high-quality RWE can reduce research costs, speed up time-to-insight, and help identify safety and effectiveness signals earlier in the product life cycle. 

For innovators, RWE should be seen as an opportunity to bolster the value proposition of a new treatment to all key stakeholders, including FDA, physicians, and payers.

Real-world evidence will not replace clinical trials. But it will increasingly determine which innovations obtain regulatory clearance, secure payer reimbursement, and reach more patients sooner. Looking ahead, the ability to measure the impact of various treatments in real-life conditions may become one of the defining features of modern healthcare.