Real-world end points extracted from administrative claims and electronic health records were generally consistent with each other and with outcomes observed in randomized clinical trials examining PD-1/PD-L1 inhibitors in patients with non-small cell lung cancer, according to recent data.

“The unprecedented availability of real-world data (RWD), emergence of new RWD sources, improved analytic methods, and the accelerating need for clinical evidence in the face of constrained RCT resources has increased the demand for real-world evidence (RWE),” the study researchers wrote. “However, before RWD finds widespread use as an adjunct to — or in unique settings, an alternative for — RCTs, the validity of readily extractable clinical outcomes measures — real-world end points—must be established.”

To do that, researchers from 6 organization used data from administrative claims and electronic health records to assess real-world end points, including overall survival (rwOS). Data sets included information from a range of 269 to 6924 patients, depending on the end point, who were treated with anti-PD-1/PD-L1 therapies between January 2011 and October 2017.

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“All data partners were able to collect information on treatment and mortality data and rapidly assemble this information to quantify real-world treatment duration and rwOS; however, information that confirmed death, including date and cause of death, varied by data source and proved to be challenging,” the researchers noted.

Analysis showed that correlations between real-world intermediate end points, such as real-world time to treatment discontinuation and time to next treatment, and rwOS were moderate to high, ranging from 0.6 to 0.9.

rwOS times ranged from 8.58 months to 13.50 months; at 1 year post–PD-L1 initiation, rwOS ranged from 40% to 57%. These medians fell into the range of median overall survival observed in relevant clinical trials.

“The implication of these findings is that RWD can provide useful and timely evidence to quantify the benefits and risks of new cancer treatments used in real-world settings,” the researchers wrote. “These results further demonstrate the utility of RWE and the need for additional investigations to assess readily extractable end points from RWD sources.”


Stewart M, Norden AD, Dreyer N, et al. An exploratory analysis of real-world end points for assessing outcomes among immunotherapy-treated patients with advanced non-small-cell lung cancer.  JCO Clin Cancer Inform. 2019;3:1-15.