Friends of Cancer Research (Friends) recently published initial findings from its real-world evidence (RWE) pilot project.
The findings, published in JCO Clinical Cancer Informatics, demonstrate that real-world endpoints “are generally consistent with each other and with outcomes observed in randomized clinical trials, which substantiates the potential validity of real-world data to support regulatory and payer decision making,” per the report.
“Differences observed likely reflect true differences between real-world and protocol-driven practices,” concluded the paper authors, including Nancy Dreyer, chief scientific officer and senior vice president of real-world solutions at Iqvia, which joined the project in 2018 to evaluate the ability of real-world data to estimate real-world endpoints in lung cancer.
“Understanding the importance of demonstrating the validity of real-world evidence, particularly in oncology where there are so many unmet needs and complex treatments, Iqvia embraced this as an opportunity to validate the use of RWE as an efficient means to innovate drug approvals and to advance personalized medicine,” Dreyer told us.
While the initial project demonstrated the ability to extract several real-world endpoints from various datasets, the next phase aims to further characterize the role of these endpoints.
“The goal of the 2.0 pilot is to understand where and how RWE can be used to evaluate treatment effectiveness in lung cancer,” she explained.
Other participants include Aetion, the American Society of Clinical Oncology (ASCO), CancerLinQ, Cancer Research Network, Cota, the US Food and Drug Administration (FDA), Flatiron Health, Mayo Clinic, McKesson, NCI SEER-Medicare Linked Database, OptumLabs, Syapse, and Tempus.
RWE: Where and when?
To implement the 21st Century Cures Act, the US Food and Drug Administration (FDA) “needs to know where and when real-world data can be trusted,” said Dreyer, adding that the regulator also needs to determine when one or more traditional randomized trials need to be conducted.
“As a by-product of this work, we are learning how RWE differs from evidence that comes from the idealized situations studied in traditional trials,” she said. “RWE is complementary to randomized clinical trials because the way patients are cared for in a trial is not the same as in routine care, and therefore the outcomes could be different as well.”
Next steps will involve characterizing the similarities and differences in data sources and their covered populations, explained Jennifer Christian, vice president, Center for Advanced Evidence Generation, Iqvia.
Iqvia will use randomized clinical trials (RCTs) as benchmarks for comparison to conduct sensitivity analyses, she told us, noting the need to evaluate the framework in other therapeutic areas and countries.
As part of this, Iqvia, in collaboration with Friends and Health Data Insight in the UK, also will use Public Health England’s Cancer Registration and Analysis Service to compare data from outside the US.
Said Christian, “Although differences are expected, it will be useful to know what those differences are, and whether they are likely to be artifacts of data recording or meaningful differences in benefits and risks.”