Researchers compared the relationship between randomized controlled clinical trial efficacy data and real-world effectiveness for oncology therapies, published in Value in Health (July-August, 2017;20[7]:866-875).
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A prevalent concern among decision makers and stakeholders in oncology is the degree to which clinical trial efficacy correlates with real-world effectiveness for novel treatments. More research and robust data are needed to better understand this relationship.
Darius N Lakdawalla, PhD, Leonard D Schaeffer Center for Health Policy and Economics, University of Southern California, and colleagues conducted an assessment to measure the relationship between randomized controlled trial efficacy data and real-world effectiveness for oncology treatments as well as how this relationship varies depending on a trial’s use of surrogate vs overall survival (OS) endpoints. Researchers assessed treatment efficacy measures of 21 phase III randomized controlled trials reporting OS and either progression-free survival (PFS) or time to progression endpoints. Cancer types included in the trials were breast, colorectal, lung, ovarian, and pancreatic. Real-world OS was estimated as the mortality hazard ratio among patients meeting randomized controlled trial inclusion criteria in Surveillance and Epidemiology End Results Medicare data.
Cox proportional hazard regression model was used to evaluate the differences between real-world mortality hazard ratio and the hazard ratios on the basis of randomized controlled trials using either OS or PFS/time to progression surrogate endpoints.
Results of the study showed that treatment arm therapies reduced mortality in randomized controlled trials relative to control arms (average randomized controlled trial mortality hazard ratio, 0.85, 0.56-1.10) and lowered progression (average surrogate hazard ratio, 0.73, 0.43-1.03). For those real-world patients who used either the treatment or the control arm regimens, real-world mortality hazard ratios were 0.6% higher than randomized controlled trial mortality hazard ratios. Additionally, real-world mortality hazard ratios were found to be 15.7% higher than randomized controlled trial surrogate hazard ratios.
Authors of the study concluded that real-world OS treatment benefits were similar to those reported in randomized controlled trials based on OS endpoints. However, real-world treatment benefits were almost 16% less than efficacy estimates based on surrogate endpoints from randomized controlled trials.
Results varied by tumor type and line of therapy, authors acknowledged.—Zachary Bessette
