A panel of experts at AMCP 2025 explored the expanding role of real-world evidence (RWE) in health care decision-making, offering practical strategies for obtaining and assessing real-world data (RWD), generating high-quality RWE, and the potential for applying it across the drug development lifecycle to inform regulatory, clinical, and payer strategies.

“We know that payers are very much interested in using RWE. But we also know that not many players are incorporating RWE into their decision-making processes yet,” said the panel. 

The panel explained that RWE generated from RWD is transforming health care by bridging the gap between research and everyday practice using data collected from patients' health status and routine health care delivery. On the other hand, RWE represents clinical insights derived from analyzing RWD about the usage and potential benefits or risks of a medical product. Data sources include electronic health records (EHRs), administrative claims, registries (product or disease), digital health technologies, patient-reported information, chart reviews, surveys, and prospective observational studies. 

While randomized clinical trials (RCTs) have long been the gold standard for evaluating drug therapy based on safety, efficacy, and tolerability, RWE is increasingly being recognized as a valuable complement for real-world decision-making. Limitations in RCTs often leave significant evidence gaps, resulting in coverage decisions made without the full body of evidence. Advances in methods, data availability, and quality allow RWE to inform treatment and coverage decisions based on the realities of clinical practice. 

As regulatory bodies like the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) increasingly consider RWE to inform their regulatory decisions, including post-market safety monitoring and drug approvals, its national and global significance continues to grow. Most pharmaceutical companies are investing in RWE, creating opportunities for payers to incorporate RWE into formulary and medical policy decision-making. Furthermore, payers are interested in using RWE as part of their decision-making process but express the need for more training on how to assess the quality of RWD for research purposes, evaluate RWE studies, and practically apply it to their decisions. 

The panel shared data from the IQVIA RWE Leadership Forum that identified 5 barriers to increased utilization of RWE from a payer perspective. These included a lack of common guidelines, a lack of expertise in data assessment and analysis, a lack of trust in pharma-sponsored RWE studies, a poor understanding of RWE best practices, and challenging outcome definitions and data collections for outcomes-based agreements. 

“Understanding and addressing these challenges will be the first step in paving the way to unlocking the full potential of RWE health care decision-making through better adoption in future payer value assessments,” shared the panel. 

A framework for RWE evolution across the product lifecycle, criteria defining good RWE, and payer education about RWE were all noted as necessary to drive greater use of RWE in decision-making. 

Through the Inflation Reduction Act (IRA), the Centers for Medicare & Medicaid Services (CMS) will negotiate drug prices for certain high-spend products that are 7 years old (small molecule) or 11 years old (biologics). Of the many factors that go into influencing negotiations, the panel noted areas where RWE can play an important role, such as the comparative effectiveness of a drug in specific populations. 

“If there is impetus to now use RWE, does RWE exist for CMS to use in order to inform IRA price negotiations?” the panel posited. 

To answer the question, in 2023, they reviewed the availability of comparative effectiveness RWE research in Medicare patients for the 10 drugs set to undergo CMS price negotiations in 2026. Of the 170 real-world comparative studies identified, 55 used Medicare RWD while 34 used commercial claims data in conjunction with either Medicare Advantage or Medicare Supplementary databases. 

Studies using RWE can be used in descriptive or comparative study designs, and panelists highlighted the many diverse options for RWE use in research. 

“This is a really important way that we can supplement and add to the totality of evidence,” said Ashley Jaksa, MPH, vice president of science at Aetion. 

When comparing the use of RCTs and RWE in studies, the biggest difference noted was randomization. Since RWE studies are observational and noninterventional, they lack randomization. RWE also tends to have more diverse populations, variable treatment, can be less expensive, and more. While RCTs may be the gold standard for causal relationships, RWE has strengths such as high generalizability, long-term safety and effectiveness, applicability to rare diseases, and feasibility when RCTs are not practical or ethical. However, RWE also has limitations such as being subject to biases that require professional intervention to mitigate and can weaken perceptions of trust. 

“RCT and RWE should not be opposed but viewed as complementary approaches. RWE allows decision-makers to broaden existing evidence and make better health care decisions,” emphasized the panel. 

RWE data quality must be assessed for accuracy, completeness, provenance, and transparency of data processing. Data relevance is determined through the availability of key data elements, representativeness, sufficient subjects, and longitudinal data capture. 

“We’ve been hearing payers over the last few years saying, ‘I really want RWE that reflects my population’. Increasingly in the US, we have EMR data that is going to be available for research. But it really requires you, as a pharma company, to understand the payer you are presenting the data to,” said Elizabeth Powers, MBA, vice president and general manager of regulatory science and study innovation at IQVIA. 

Reference 
Jaksa A, Powers E, Sweet B. Education Session: [R1] Unlocking the Value of Real-World Evidence (RWE): Insights for Payers and Manufacturers. Presented at: AMCP 2025; March 31-April 3; Houston, TX.