Real-World Evidence

Real-World Evidence in Healthcare: A Complete Guide

In today's evolving regulatory landscape, clinical researchers are turning more frequently to real-world evidence to complement data generated from randomized controlled trials (RCTs). With the passage of the 21st Century Cures Act, the U.S. Food and Drug Administration (FDA) acknowledged a larger role for the use of real-world data and real-world evidence to support regulatory decision making.¹

Real-world evidence is a key focus area for Veradigm. That's why we've compiled this comprehensive guide to real-world evidence, complete with links to use-cases, relevant blog articles, white papers, and more.

What Is Real-World Evidence?

Real-world evidence (RWE) is the evidence derived from real-world data. RWE is clinical evidence regarding the use, potential benefits, and/or risks of a medical therapy. This type of evidence can be gathered by examining data from a variety of different types of research such as observational studies (both prospective and/or retrospective), randomized trials, large simple trials, and pragmatic trials.²

Why Is Real-World Evidence Important?

Real-world evidence is important because it can often provide a more comprehensive understanding of how a new therapeutic option will work in the “real world” rather than via standard RCTs alone. While RCTs remain the gold standard in the approval of new medical therapies, they often cannot provide the full picture.³ A standard RCT enrolls a small segment of the disease population and tests therapies in a controlled environment. What's more, RCTs are costly and take a considerable amount of time to complete.

By using real-world evidence, we can enhance our understanding of what works for different patient types in greater context. Real-world evidence allows researchers to examine the performance of drug treatments and other interventions while also looking at other factors and variables. In addition, RWE generation is more cost effective and can happen more quickly than standard RCTs.

RWE can generate insights into how treatments perform among specific patient sub-groups that may have not been studied in the RCTs. For example, with RWE, researchers can study how new therapies work among patients with co-morbidities, certain age groups, or specific socio-demographic groups. Real-world evidence can also help researchers understand what happens to a patient over a lifetime, not just throughout the RCT period.

Because the insights gleaned from RWE can be so powerful, the FDA uses real-world data and RWE to monitor post-market safety and adverse events, and in support of regulatory decisions.²

How Is Real-World Evidence Used?

Real-world evidence can be widely used throughout the healthcare system. From the physician’s office, to life science research, to health plans and payers, policy makers, and regulators, RWE can make an impact across the spectrum.

Life Science Research

Life science researchers and biopharmaceutical companies are significant stakeholders when it comes to real-world evidence. As we’ve mentioned, real-world evidence can complement the findings of traditional RCTs. RWE can provide life science researchers with additional insights into the broader impact of a medication’s use in routine clinical care, including the safety and efficacy of novel therapies.

Policy Makers and Regulators

The use of real-world data and real-world evidence can help regulators monitor post-market safety and adverse events, and aid in FDA decision-making.² Therefore, utilizing real-world evidence has the potential to impact medication approvals and accelerate the drug development process. Further, the Centers for Medicare and Medicaid Services (CMS) use RWE to design value-based contracts and to evaluate innovation models for Medicare and state Medicaid programs.⁴

Health Plans and Payers

Health plans and payers also realize the value of real-world evidence. The value spectrum payers perceive from real-world evidence includes informed safety monitoring, utilization management, and cost/value analysis. While it varies from organization to organization, some see benefit in utilizing real-world evidence studies to inform pharmacy and therapeutic (P&T) committee decisions.5 While high-quality RCTs remain critical in payer decision-making, some payers are beginning to turn more frequently to RWE to for greater validity.

Additionally, payers are turning to real-world evidence to evaluate value-based care outcomes including the use of medicines. In some cases, payers are tying payment for treatment to both short and long-term effectiveness of pharmaceuticals and other therapies.⁶

Physicians and Healthcare Providers

The application of RWE for clinicians is somewhat more indirect than it is for other healthcare stakeholders. Primarily, healthcare providers can make use of clinical guidelines derived from RWE. The findings generated by real-world evidence can aid in a care team's understanding of a disease and help guide data-driven treatment decisions.⁷ Because real-world evidence has the potential to complement findings by adding perspective to the results of RCTs, RWE can empower physicians to make more targeted and appropriate treatment plans for their patients.

Real-world evidence is only as good as the real-world data used to generate it. Physicians and other healthcare providers are a critical part of the data gathering process. During an encounter with a patient, clinicians routinely use electronic health record (EHR) systems to enter structured clinical data such as diagnoses, lab tests ordered with results, and medications prescribed. Additionally, providers submit claims to payers that offer another source of real-world data. These data can then be de-identified and used to generate real-world evidence.

If you are interested in learning more about real-world data, check out our comprehensive guide for a more in-depth look.

How Is Data Used In Real-World Evidence?

As we’ve mentioned, real-world evidence is generated by real-world data. Real-world data come from a variety of sources including:

• Electronic health records (EHRs);
• Claims and billing activities;
• Product and disease registries;
• Data gathered from other sources such as mobile devices, wearables, etc.¹

The analysis of real-world data can generate valuable insights in the form of real-world evidence. Technological advances and the ubiquity of digital data collection have created a wealth of real-world data. Through robust, efficient, and accurate analysis, researchers can harness the power of this data into truly transformative action.

If you are interested in learning more about real-world data, check out our comprehensive guide for a more in-depth look.

What Is The Difference Between Real-world Evidence and Real-World Data and How Do They Work Together?

Occasionally the terms “real-world evidence” (RWE) and “real-world data” (RWD) are used interchangeably, and while RWE and RWD are undoubtedly connected, they are distinct concepts. In this diagram, we've broken down the basic relationship between RWD, RWE, and their applications in the healthcare system.

In truth, the interplay between RWD, RWE and their applications in the healthcare system are much more complex and require a great deal expertise to navigate. To see how Veradigm utilizes real-world data and real-world evidence to gain valuable healthcare insights, check out some use-cases linked below.

Real-World Evidence and Veradigm

Veradigm helps organizations achieve real-world insight from the point of care throughout the patient journey. Our innovative solutions allow our clients to discover timely, actionable real-world evidence to improve patient experience and outcomes, and can help you demonstrate how your products can benefit patient health.

Access to our expansive commercially available ambulatory EHR dataset helps our clients leverage de-identified data through innovative analytic technology for outcomes research. With Veradigm, you can execute actionable retrospective analyses and prospective programs, all with the support of a dedicated research team and custom data visualizations and dashboards so that you can better understand the patient journey from data directly captured at the point of care.