Assessment of quality and clinical significance of endpoints in cancer immunotherapy
Journal Title: JOURNAL OF HEALTH POLICY & OUTCOMES RESEARCH - Year 2015, Vol 8, Issue 2
Abstract
This review describes available immunotherapeutic agents approved for the treatment of prostate cancer (sipuleucel-T), advanced melanoma (ipilimumab, pembrolizumab, nivolumab) and NSCLC (nivoluamb) and underline that their specific mechanism of action require to use appropriate endpoints for the efficacy evaluation. The FDA and EMA guidelines on endpoints in clinical trials indicate the use of overall survival as a primary endpoints. However, there is a trend for using the time-to-event endpoints for drug approval since 1990. Oncological clinical trials utilize apart from OS also the endpoints based on tumor assessment – e.g. progression-free survival, disease-free survival or response rate. This review presents the differences in mechanism of actions between standard chemotherapy and immunotherapy which imply the significant differences in the kinetic of response and long-term effects. The WHO and RECIST response criteria were developed to estimate the effect of cytotoxic drugs on cancer and the new patterns of response observed after treatment with immunotherapeutic agents indicate the need for adopting novel criteria in the evaluation of tumor responses. The performed review of pivotal clinical trials assessing the efficacy of immunotherapy showed that the most commonly evaluated endpoints were: OS, PFS and RR. Prolonged survival with concomitant lack of benefit in PFS was explained by the need for applying irRC for evaluation of the efficacy of immunotherapeutic agents beyond the classical measurement. It could be concluded that trial design which takes into account disease characteristics and immunotherapeutic agents’ mechanism of action is the key to define appropriate endpoints and proper evaluation of the efficacy.
Authors and Affiliations
Katarzyna Miernik, Ilona Czarny-Ozga, Jacek Walczak
Keywords
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- EP ID EP192166
- DOI 10.7365/JHPOR.2015.2.5
- Views 81
- Downloads 0
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