Real-world data

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Clinical trial data remains the ‘gold standard’ for demonstrating the effectiveness of an intervention and seeking regulatory approval. But in October 2021 the FDA conceded that the health care community uses real-world data to support expanded coverage decisions and to develop guidelines and decision support tools for use in clinical practice. It acknowledged that real-world data and the resulting real-world evidence was playing an increasing role in health care decisions and offering complementary insights.

Real-World Data

Real-world data is collected from electronic health records, electronic medical records and national registries. Patient-reported outcomes, app-based questionnaires from mobile devices, wearables and patient surveys all add to the trove of information that is now available to every practitioner. Nancy Dreyer of IQVIA said,  “Randomized controlled trials are considered the gold standard of evaluating whether a medical treatment CAN work, but not whether the treatment DOES work for diverse patients in real-world situations.”

To utilize real-world data effectively it is important to have a thorough understanding of the data source and how specifically relevant it is to the research question being addressed. This analysis can help doctors make more informed treatments decisions particularly for patient subgroups that may not have been well represented in clinical trials. It can also help doctors compare the effectiveness of different treatment options for the same disease that often isn’t directly compared in a clinical trial.

Payers also benefit from real-world data by seeing which medicines can reduce healthcare costs. Clinical trials don’t demonstrate how a medicine might reduce the time in a hospital for a patient or reduces the chance of ending up back in the hospital after treatment.

Real-World Evidence

Real-world evidence is clinical evidence of the potential benefits or risks of a medical product that is derived from the analysis of real-world data. Real-world evidence can be generated through multiple study designs including randomized trials, large simple trials, pragmatic trials and observational studies. Clinical trials are conducted in experimental settings with defined patient populations while real-world evidence studies are largely conducted in real-world settings with more heterogeneous, larger patient populations.

An example of real-world evidence that complemented clinical trial data is the case of BAVENCIO (avelumab). In 2017 BAVENCIO received accelerated approval from the FDA for metastatic Merkel cell carcinoma from the findings of a single-arm, open-label, Phase II study. Electronic health record data for historic controls who met the study inclusion criteria were utilized to help characterize the natural history of the disease. Murtuza Bharmal of Merck said, “Conducting a traditional randomized controlled study was ethically challenging and not feasible from an operational perspective. The real-world evidence enabled the regulatory authorities to put the efficacy of avelumab, as measured in the single-arm trials, into context, like a historic control arm.”

The 2016 passage of The 21st Century Cures Act  has helped promote more rapid development of drugs and biologics by enhancing the FDA’s ability to modernize clinical trial design including the use of real-world data and real-world evidence.

Interested in a career in the support of this analysis? Contact Smith Hanley Associates’ Biostatistics and Clinical Data Management Executive Recruiter, Nihar Parikh at nparikh@smithhanley.com.


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