Data is the Difference: Leveraging Real-World Data and Real-World Evidence for Better Outcomes, Value

Real-world data (RWD) and real-world evidence (RWE) play an important role in helping life sciences organizations and providers understand the safety, effectiveness and value of healthcare interventions in everyday practice.

RWD usage has seen an uptick with 90 percent of global life sciences executives indicating plans to leverage actionable data for decision-making across a product’s life cycle. In addition, one study found that 116 out of 378 FDA-approved New Drug Applications (NDA) or Biologics License Applications (BLAS) incorporated RWD/RWE in the submission, with the proportion of approvals incorporating RWD/RWE increasing each year between 2019 and 2021.

However, amidst the increase in RWD/RWE usage, confusion remains about what type of data is most meaningful – and when and how RWD/RWE can be leveraged to inform the medical solution decision-making process or regulatory submissions for sustained impact and the betterment of patients worldwide.

Let’s separate fact from fiction on leveraging RWD/RWE to optimize healthcare outcomes and value.

1. Myth: RWD sources lack standardization and connectivity.

Fact: RWE is only as good as the data from which it’s sourced. To evaluate the RWE necessary to assess a product’s intent and value in the clinical setting, life sciences organizations need different sources of data, which when brought together, can generate actionable information.

The Premier Healthcare Database (PHD) is a HIPAA-compliant retrospective database filled with more than 20 years’ worth of chargemaster data that includes patient, hospital, outpatient and clinical characteristics, costs and charges, laboratory data, vitals, treatment and diagnoses. As of July 2024, the data encompasses over one billion individual patient encounters, spanning the continuum of care and includes visits from approximately 260 million unique patients.

• Data from approximately 1300 unique sites of care and more than 1.3 billion patient encounters, encompassing 45 percent of U.S. inpatient discharges, 2.7 billion outpatient and clinic encounters and 177 million physician office visits.
• Privacy-preserving record linkage technology to unite specialized data sources and clinical trial data with RWD.
• Data includes different types of coverage from commercial payers to Medicare and Medicaid across the continuum of care and including medication/device SKU-level utilization details as well as longitudinal data on mothers and their babies from prenatal to post-partum.

“As life sciences and medical device companies continue to bridge the gap between medical solutions development and real-world application, leveraging real-world data for real-world evidence generation has emerged as a game changer,” said Myla Maloney, Chief Growth Officer, PINC AI™ Applied Sciences.

2. Myth: RWD/RWE cannot enhance the “Gold Standard” of randomized clinical trials (RCTs) or support regulatory submissions.

Fact: While RCTs remain a strong tool for assessing a product’s efficacy and safety, RWD provides important insights on a product’s impact in routine clinical practice that can complement findings from RCTs and in some circumstances replace the need for additional RCTs that might otherwise be required. A key example of this is using RWD to assess the impact of a therapeutic intervention in populations not studied in an RCT.

RWE is a complex area, especially when it comes to regulatory submissions and can be a great source of confusion for providers. While it isn’t clear cut, the Federal Drug Administration (FDA) did release a framework and draft guidance to help providers make decisions regarding the use of RWD/RWE.

With the passage of the Inflation Reduction Act (IRA) in August 2022, RWE is serving as an important tool for life sciences and medical device companies striving to demonstrate the value of their products and help patients get access to the therapies and interventions they need faster.

Thanks to the 21st Century Cures Act, more regulatory agencies are recognizing the promise of RWD/RWE. When combined with clinical trial research, it has the potential to shorten the regulatory approval process and increase the chances of U.S. Food and Drug Administration (FDA) and European Union Medical Device Regulation approvals.

“We are seeing more regulatory submissions that include RWD to demonstrate product safety and efficacy, provide therapeutic context, and support expanding label indications and guidance for treatment. A key example of this is using RWD to assess the impact of a therapeutic intervention in populations not studied in an RCT,” said Essy Mozaffari, Senior Director of Global Medical Affairs at Gilead Sciences.

One medical device company partnered with Premier Applied Sciences (PAS) to better understand their device, its safety, outcomes and benefits to patients from utilization and determine potential options for label modification.

The medical device company worked with the PAS team to utilize a robust and standardized PHD dataset to help confirm that the use of their device with expanded indications could have similar safety and positive patient outcomes. They were able to leverage PHD data to help inform their regulatory filing, ensuring that the right patients could benefit from their device.

The PAS impact: By using RWE to support their device’s expanded indications for use, the company was able to avoid additional randomized clinical trials and reduced their launch research spend by up to $3 million.

Additionally, clinical trial sites have historically been selected solely based on experience and relationships with Principal Investigators at the site level. Often, the patients that meet the study criteria are not at those sites. Premier’s Applied Sciences (PAS) division is “flipping the funnel” to recruit for clinical trials based on where patients are versus where the relationships exist.

PAS’ robust RWD and AI-enabled solutions are estimating eligible patient population volumes with a new level of granularity and efficiency previously unattainable when screening sites to match trial protocols.

Today, a clinical trial for a chronic condition is utilizing Premier’s collaboration with Datavant to link clinical trial patient data and PHD data to understand the participants’ complete patient journey. By uniting clinical trial data with real-world data contained in the PHD, researchers are gaining insight into medication history, which can be difficult to collect, as well as the ability to follow the patients forward after the trial has ended, when oftentimes patients are lost. The real-world evidence generated from the linked data can be used to assess important long-term health outcomes outside of the clinical trial, improve drug safety and efficacy, and empower the healthcare industry to treat chronic care conditions more effectively.

What’s more? Life science organizations are leveraging RWE control arms – which may eliminate the need for researchers to construct a separate control arm – potentially shortening the duration and cost of a clinical trial. This can also help improve the patient experience and outcomes by enabling all study participants to partake in the treatment and its potential benefits rather than a placebo, thereby increasing their access to medical care.

3. Myth: RWD/RWE isn’t robust enough to aid in early disease state identification and intervention.

Fact: Life sciences organizations can partner with PAS to leverage PAS’ AI capabilities – including RWD coupled with machine learning (ML) and natural language processing (NLP) – enabling speed-to-value and a data-driven understanding of disease progression, early disease identification and intervention.

NLP can read more than two million records per hour, identify trends and understand unstructured information contained within clinicians’ free-text notes, pathology reports and genomic results to help deploy evidence-based guidance at the point of care.

By uniting clinical trial data with RWD contained in the PHD, researchers can gain insight into medication history, which can be difficult to collect, as well as the ability to follow the patients forward after the trial has ended, when oftentimes patients are lost. The RWE generated from the linked data can be used to assess important long-term health outcomes outside of the clinical trial, improve the processes for evaluating drug safety and efficacy, and empower the healthcare industry to treat chronic care conditions more effectively.

The PAS impact: The PHD dataset and RWD/RWE helped one medical device company prove that its device, with expanded indications, was substantially equivalent to using the predicate device, thus clearing their product of further RCTs and testing.

RWD/RWE is Crucial for the Future

RWD/RWE is changing how life sciences organizations develop, discover, commercialize and receive reimbursement for drugs and devices.

And the RIGHT RWD is key – robust, high-quality data can help generate RWE that provides insights into how clinicians are treating patients and what outcomes are being realized in practice across the United States. When coupled with AI-enabled technology, life sciences organizations can streamline financial, clinical and operational processes to expedite product development at reduced costs for clinicians and the patients they serve.

To get the most benefit out of RWD/RWE, finding the right partner is critical, requiring access to broad, standardized and clean datasets, research-ready testing networks, and AI-enabled solutions for speed to value.

Learn more about Premier Applied Sciences and how you can use the Premier Healthcare Database to conduct evidence-based analyses of drugs, devices and disease states and accelerate medical solution development.