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Real-World Data for Clinical Evidence Generation in Oncology Sean Khozin, MD, MPH

What is real-world data (RWD)?

RWD is data collected at the point of routine care or directly from patients outside of conventional clinical trials (CCTs) from sources such as electronic health records (EHRs), insurance claims, patient registries, and digital health solutions such as biometric sensors. A definition that focuses on the original intent behind collection of clinical data can be used to characterize the core characteristics of RWD. Using this framework, purposeful collection of data at the point of care for research, rather than routine delivery of health care services, narrows the gap between real-world and CCT data, providing a foundation for optimal experimental designs that include randomization at the point of care (ref):

The intended use of point of care data at the time of collection is the primary feature informing potential use cases of real-world data for clinical evidence generation

What are some of the problems with CCTs in oncology?

1. CCTs can be slow and costly (ref, ref):

Estimated average per-patient clinical trial costs, by selected condition, 2013
Clinical trial costs by therapeutic area and phase (in US $ million)

2. CCTs often produce results with limited external validity, which is a measure of generalizability of the results to real-world patients (ref).

"Historically, researchers have tended to focus on maximizing internal validity, with the idea that it is more important to know if a given public health intervention works under highly controlled conditions than it is to know if it will work among different population groups, organizations, or settings. Similarly, funding organizations and journals have tended to be more concerned with the scientific rigor of intervention studies than with the generalizability of results. The consequence of this emphasis on internal validity has been a lack of attention to and information about external validity, which has contributed to our failure to translate research into public health practice" (Steckler A, et. al.)

Main issues that can affect external validity of CCTs

3. Less than 5% of adult oncology patients (especially minorities, the elderly, low-income individuals, and those living in rural areas) have the opportunity to participate in CCTs because of barriers that include financial and strict exclusion criteria.

"In oncology trials, specific populations are often categorically excluded without a clear reason." (Beaver J, et. al.)

How can RWD address the deficits of CCTs?

RWD can be used to improve current methods of clinical evidence generation, enabling study designs that produce results with an appropriate balance between internal and external validity. Real-world clinical studies can be both retrospective and prospective. Prospective collection of RWD can enable evidence generation based on what is often called pragmatic clinical trials (PCTs). PCTs can fully leverage the technical innovations of modern times (e.g., EHRs, and digital health solutions) to support experimental designs such as randomization while addressing growing societal demands for access to experimental therapies by seamlessly incorporating clinical research into routine delivery of care in a cost-efficient and patient-centered manner.

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Sean Khozin
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Created with images by Sebastien Gabriel - "Abstract cubes artwork"

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