Can real-world data enrich clinical trials?

COVID-19 urgently highlighted the need for real-time data, and with it, the need to tap into a wider variety of data sources to answer clinical questions has become abundantly clear. Traditionally, clinical trial data is gathered by participating researchers themselves according to the study protocol, regardless of the availability of relevant “outside” information that might enhance clarity of the findings. Therefore, in conventional clinical trials, useful information about the participating patients, such as prior real-world disease and treatment experiences, are necessarily omitted. Additionally, many trials don’t routinely continue to follow participants’ progress after the active treatment/evaluation period of the study.

Today’s data picture is excitingly different, as electronic health records, device data and patient-generated data enable us to learn so much about the prospective and retrospective patient journey. Real-world data and real-world evidence derived from sources that go beyond those of the individual participants in a clinical trial enhance the conclusions we can draw from trials, better guiding and influencing patient outcomes.

According to FDA, RWD is information relating to patient health status and/or the delivery of health care which is routinely collected from a variety of sources including electronic medical records, medical claims and billing, product and disease registries, mobile devices and from patients themselves. RWE, by contrast, is the clinical evidence about the use and potential benefits or risks of a medical product derived from analyzing RWD. RWD sources can be used to collect data and may allow for developing an analysis infrastructure to support many types of clinical trials.

Using real-world data (RWD) and real-world evidence (RWE) to enhance clinical insights in clinical trials

Combining or contrasting data from traditional clinical trials with real-world data can garner many novel insights. By merging these siloed data points, and augmenting them with incremental licensed data, opportunities open up to learn more about patients pre- and post- study as well as during the active study period. How does the data tell this patient journey story? We begin with patient consent, allowing the collection of data outside the realm of study data. Once consent has been obtained, data must be de-identified from sources such as payers or pharmacy claims or hospital chargemasters. And then the data needs to be linked across these disparate sources to ensure that all transactions belong to the same individual regardless of variances such as the number of times they may have moved, or changes in insurance providers. And finally, this data needs to be linked to study data. All in a HIPAA-compliant manner.

HealthVerity solutions make these scenarios easily possible, granting study investigators the ability to evaluate transactions relating to the patient retrospectively, before the study began, in real-time during the trial, and even prospectively after the trial concludes, to understand long-term ramifications. Our technologies grant the ability to evaluate retrospective patient data on study subjects upon enrollment, for example, gather real-time insights on efficacy and adverse effects during the trial, potentially link data from other trials, and afterwards, continue to follow up on how the patients are doing in the real-world, even beyond the limited parameters of extension arms. No patient is ever really “lost to follow up” with this unique ability to gain further insights. In addition, RWD allows for creating a “synthetic or observational arm” of a clinical study using data from a pool of relevant subjects who are not part of the enrolled trial participants. This can help with decision-making in multiple ways, including the feasibility of running additional trials to expand a treatment population. The following list shows just some of the possibilities afforded by adding real-world data to a well-designed clinical trial.

Key uses of RWD:

• Updating side effect rates post-launch
• Discovery of previously unknown side-effects
• Single-arm experimental trials
• Digital approvals
• Biosimilar development
• Improved patient selection
• Efficient data collection
• Assessing the gap between reported and clinical efficacy
• Efficacy in specific populations
• Post-trial long-term outcomes
• Establishing standards of care
• Evaluating the effect of payors on care
• Clinical decision support from the perspective of physicians, patients, and the community  setting

An important aspect of RWE relevant to today’s concerns, especially in the era of COVID-19, is the ability to improve the diversity of study subjects and researchers. Historically underrepresented groups, such as people of color, people with language barriers, or those with distrust of the healthcare system can now be reached and included in a culturally sensitive way. By building diversity into trials from the conception stage, research and marketing teams can receive the benefits that ensue from testing new drug therapies in the widest possible array of human beings—and including those who in particular may be likely real-world future users of the product—and in the past were overlooked altogether when trials were designed.

The FDA’s RWE Program under the 21st Century Cures Act is ongoing, evaluating the potential use of RWD to generate real-world experience of product efficacy that can support new indications of existing drugs and biologicals, as well as supporting requirements for post-approval study. Under this mandate, the HealthVerity Provider Diversity Index can address FDA guidance on minority inclusion in trials, identify investigators with more diverse patient populations and highlight physicians who are more likely to treat minority patients at higher risk.

Partnering with HealthVerity

HealthVerity’s solutions provide the infrastructure our clients need to leverage the enhanced learnings RWD can provide. Based on the foundation of identity, privacy, governance and exchange, there is no faster or more accurate way to de-identify, match and link health data. Our solutions are comprehensive, managing consent, licensing RWD via HealthVerity Marketplace, through delivering real-time identity matching and resolution technologies directly into the workflow with 10x greater accuracy, found nowhere else. HealthVerity Marketplace is the largest real-world data ecosystem, encompassing over 75 healthcare and consumer data sources, 150 billion transactions and 330 million US patients.

The role played in today’s healthcare marketplace by real-world data and real-world evidence will only expand in the coming decades, along with the need to work with a partner with expertise in maximizing its value.