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Advances the reliability of RWE for regulatory decision making

Cardinal Health’s Real-World Evidence and Insights team has completed research designed to advance the reliability of real-world evidence (RWE) for regulatory decision making. The research was funded by the U.S. Food and Drug Administration (FDA), which awarded the RWE team a $750,000 contract in 2021. With the funding, Cardinal Health evaluated real-world data (RWD) for lymphoma tumor response assessment compared to blinded independent central review, the gold standard in clinical trials. The novel method is called real-world Lugano (rwLugano) and corresponds to Lugano 2014, the criteria used in clinical trials to classify lymphoma treatment response.

The use of RWE may ultimately support patient access to safe and effective therapies, but only if the potential is fully realized by bridging the measures used in clinical trials to corresponding endpoints in real-world patients. The Real-World Evidence team completed their research this year, finding that their rwLugano method of measuring response to lymphoma therapy appeared more accurate than the standard approach used in most studies.

“The goal of our study was to improve what is widely regarded as the best real-world measure of response to therapy – which is physician-charted response, as recorded in a patient’s medical records,” said Scott Swain, PhD, MPH, senior director, Real-World Evidence & Regulatory Science. “The study’s objective was to see if our new measure was more similar to the clinical trial standard, which is a blinded independent central review, than the physician-charted response. We proposed to build an algorithm comprising the same clinical features used in centralized review to get closer to the clinical trial standard.” 

RWE is the clinical evidence about the use and potential benefits or risks of a medical product derived from analysis of RWD, which the FDA defines as data about patient health status and/or the delivery of healthcare that is routinely collected from a variety of resources, such as data from electronic health records or medical claims data. The RWD for this study was abstracted from patient medical records via a physician-led chart review.

RWE varies from clinical trials, which are the gold standard for determining medical product safety and efficacy and gaining FDA approval. Clinical trials are done in controlled environments to assess various therapeutic interventions. They’re built on rigorous protocols using carefully screened volunteers. The volunteers tend to be younger, less diverse, and healthier than the population as a whole – and therefore, their results may not reflect medical care for those patients outside the study. A patient’s age, race, gender, ethnicity and other personal, cultural and environmental factors can cause the patient to have a different experience to the same treatment from the clinical trial volunteers.

With RWD, researchers may have access to a larger sample size than  the clinical trial. They also might be able to target patients who are harder to find for a clinical trial, as clinical trials are often conducted in a limited number of academic centers. Another advantage of looking at RWD is that researchers can observe drug utilization and treatment choices in a representative population in routine clinical practice. Using RWD allows researchers to observe real-world medical practices, treatment patterns, long-term safety and effectiveness, and to evaluate rare adverse events.

In this research study, the Cardinal Health team looked at lymphoma patients’ response to therapy and separately compared the response recorded in the patients’ medical records, and their novel rwLugano algorithm to the clinical trial standard – blinded independent central review per Lugano 2014. The study findings suggest that the rwLugano algorithm was more similar to the clinical trial standard than the physician-charted response.

The study demonstrated that robust methods using RWD may accurately replicate lymphoma treatment assessments, increasing compatibility between RWD and clinical trial data. The team is looking to further validate this finding as well as similar outcome assessment methods in solid tumors. Using such methods to measure patient outcomes could support regulatory approvals, facilitating patient access to novel therapies. Ultimately, this study, and others like it, may lead to improvements in lymphoma RWE research, which may include regulatory objectives.

“Cardinal Health has unique relationships with physicians that allows us to do this type of research,” said Andy Klink, PhD, MPH, senior director, Real-World Evidence & Insights. “Many other RWD sets rely on researchers to curate or abstract data. We work directly with treating physicians, which allows us to get the deepest clinical information and the right data. Sometimes when researchers look at patient medical records, they rely on what is explicitly charted, whereas our network physicians know how to navigate and interpret their patients’ medical records in ways that other researchers might miss because relevant clinical data is often found in unstructured fields.”

Aklilu Tedla, JD, vice president and general manager, Real-World Evidence & Insights, said, “Everyone involved in healthcare, be they doctors, regulators, caregivers, or patients, wants the same thing – to get safe and effective therapies to patients as quickly as possible. This study represents our commitment to improve the state-of-the-art to provide better RWD and generate improved RWE that leads to improving available evidence to support regulatory approvals and better care.”

About our RWE experts: Cardinal Health’s Real-World Evidence and Insights team generates RWD and RWE to arm pharmaceutical manufacturers with insights to help them make strategic decisions for their products. The data are also used by various stakeholders to assess safety and effectiveness of a product to inform treatment decisions for physicians and patients; formulary decisions for payors; and to support regulatory decisions for the FDA.  Learn more about this work here.