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Assessing the efficacy-effectiveness gap for cancer therapies: A comparison of overall survival and toxicity between clinical trial and population-based, real-world data for contemporary parenteral cancer therapeutics

Cancer Jan 29, 2020

Phillips CM, Parmar A, Guo H, et al. - Researchers sought to quantify the magnitude of survival and toxicity differences that exists between clinical trial (CT) and real-world evidence (RWE) for contemporary cancer systemic therapies. They recognized patients who were administered cancer therapies funded under Cancer Care Ontario's New Drug Funding Program (NDFP). Recognition of Landmark CTs with data concerning survival and adverse events (AEs) for each drug indication was done. Canadian population-based databases were queried for RWE for survival and hospitalization rates during treatment. From 20 systemic therapies, the inclusion of 29 indications was done. In RWE, worse survival was evident for 28 of 29 indications (97%), with a median overall survival (OS) difference of 5.2 months. Further RWE revealed lower effectiveness through a meta-analysis of an OS hazard ratio of 1.58. These findings support the evidence revealing an efficacy-effectiveness gap for contemporary cancer systemic therapies, with a 5.2-month lower median OS observed in RWE compared with CT data. Hence they recommend using RWE to adequately inform real-world decision making concerning the use of cancer systemic therapies.
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