From Supporting Role to Strategic Lever: How Real-World Evidence Is Reshaping Healthcare Decision-Making

Historically, the healthcare industry has viewed real-world evidence (RWE) as a secondary asset — whether to complement randomized clinical trials (RCTs) or provide post-market context.

That framing has shifted in recent years. Regulators are signaling greater openness to its use, life sciences and medtech organizations are investing in it at scale, and the industry is beginning to treat it as a core component of scientific evidence. Furthermore, the Food and Drug Administration (FDA) and other regulatory agencies, such as the European Medicines Agency (EMA), have continued to expand both guidance and published examples showing how RWE can inform regulatory outcomes across the product lifecycle, including in premarket applications.

But this shift isn’t happening in a vacuum. It’s being driven by a convergence of pressures and opportunities — and a growing recognition that traditional approaches alone can’t keep up.

Factors Fueling the Growing Role of RWE in Regulatory Submissions

Several forces are reshaping how evidence is generated, evaluated and used in decision-making.

1. Regulatory expectations are evolving.

Regulatory agencies are increasingly accepting high-quality RWE for certain use cases, including post-market surveillance, safety monitoring, label expansions and supplemental evidence generation. This has elevated RWE from a nice-to-have to a strategic component of the evidence base. 

2. Clinical trials don't reflect the true complexity of real-world care.

RCTs are designed to establish efficacy under controlled conditions, but they don't always account for patients with multiple conditions, diverse demographics or complex treatment histories. RWE helps stakeholders understand how therapies perform in the broader population that could receive care. Furthermore, RWD and RWE are changing how medical device companies discover new product targets and identify opportunities for repurposing existing medical solutions. The FDA has documented numerous examples of RWE applications throughout the product lifecycle, demonstrating how routinely collected data can serve as valid scientific evidence. 

3. The demand for quick evidence is higher than ever.

The life sciences industry is facing growing pressure to make decisions quickly. RWE can help accelerate speed to market: Rather than waiting for new studies to run their course — which can sometimes take years — life sciences companies can leverage RWE to evaluate treatment effectiveness, utilization patterns, outcomes and more.

4. Value-based care requires proof of real-world outcomes.

As healthcare shifts from volume to value, stakeholders need evidence that treatments improve outcomes and reduce costs in routine practice, not just clinical trial settings. RWE helps demo clinical value, economic impact and population-level effectiveness.

5. The volume and breadth of data have expanded significantly. 

Healthcare systems generate vast volumes of real-world data across clinical, operational and administrative domains. When these data sources are brought together and effectively linked, they create a longitudinal view of the patient journey from admission through discharge and beyond, providing both clinical and contextual insight into how care is delivered in practice. 

Integrated datasets such as the Premier Healthcare Database (PHD) demonstrate how this breadth and depth of data can be used to generate meaningful, actionable evidence at scale — an important advantage compared to smaller datasets representing one or two health systems. By combining multiple data types, including clinical, claims, and chargemaster data, the PHD enables analyses that extend beyond traditional data sources and more accurately reflect real-world care.

6. Healthcare questions are becoming more complex. 

Stakeholders increasingly need to address questions that traditional trials are not designed to address, such as "How does a therapy perform across different patient populations?" and "How does a treatment compare to alternatives in routine practice?"

When combined with clinical trial research, RWE has the potential to shorten the regulatory approval process and increase the chances of FDA and European Union Medical Device Regulation approvals.

But not all data — and not all evidence generated from it — is equal.

How Premier Helps Organizations Leverage RWE as Scientific Evidence

Let’s start here: RWE is only as good as the data from which it’s sourced. Life sciences companies need different sources of data that, when united, can deliver accurate, actionable information. And the right RWD is key – robust, high-quality data that fuels evidence-based and population-based analyses of drugs, devices, other treatments, disease states, epidemiology, resource utilization, healthcare economics and clinical outcomes.

To get the most benefit out of RWD/RWE, finding the right partner is critical. For Premier, the role is not simply to provide data. It’s to help advance how RWE is generated, validated and used in regulatory decision-making.

That starts with the PHD. A HIPAA-compliant, retrospective database, the PHD comprises U.S. service-level, all-payer information on 1.5 billion outpatient visits and about 25 percent of annual U.S. inpatient admissions. And, for most data elements in the PHD, less than 1 percent of patient records are missing information. For key elements in the PHD such as demographics and diagnostic information, less than 0.01 percent are missing data.

For example, a medical device manufacturer, working with Premier to conduct a RWE-based study that included an intensive chart review with a historical control, was able to update warning language associated with one of its products. The update allowed on-label use of the device under certain conditions in the U.S. The submission represented a meaningful regulatory and competitive milestone for the manufacturer.

Regulatory outcomes depend on a variety of factors including study design, data quality and agency-specific requirements. While the use of real-world evidence does not guarantee approval or specific regulatory outcomes, the example above highlights not only what is possible but also what is required. Regulatory decisions are based on the totality of evidence and are subject to agency review. As RWE becomes more applicable in regulatory reviews, the focus must shift to ensuring data meet expectations for relevance and reliability. 

Against this backdrop, the National Evaluation System for Health Technology (NEST), an initiative within the Medical Device Innovation Consortium (MDIC), was established to support the sustainable generation and use of timely, reliable and cost-effective RWE across the medical device lifecycle, using high-quality RWD analyzed with robust methodological standards. Through the NEST Mark approach, this includes:

• Applying well-defined processes to evaluate the relevance and reliability of RWD to generate RWE.
• Helping de-risk the use of RWD for supporting regulatory filings.
• Improving confidence in the resulting evidence for consistent evaluation of RWD across the product lifecycle. 

Premier’s collaboration with NEST contributes to this work by applying its high-quality data assets in support of the NEST Mark approach for generating real-world evidence for regulatory decision-making. Together, the collaboration aims to enhance the predictability in medical device evaluations.

The Bottom Line

Regulators are asking different questions. The industry is under new pressures. And the data landscape is expanding rapidly.

In this environment, success depends on more than access to data. It depends on the ability to utilize that data, generating evidence that is:

• Credible enough for regulators.
• Relevant enough for clinicians.
• Actionable enough for business decisions.

Premier plays a central role in that evolution, helping organizations not just keep pace with the changing evidence landscape but move it forward.

Because in today’s environment, the goal isn’t just to obtain more data.

It’s to generate evidence that changes the trajectory of clinical decision-making, regulatory strategy and, most importantly, patient outcomes.

To learn more about how the right data, methodology and expertise come together to generate RWE that stands up in practice, get your copy of "Data is the Difference: Unlock the Power of RWD to Maximize Value.”