Patient Reported Outcomes in Clinical Trials Leading to Cancer Immunotherapy Drug Approvals from 2011 to 2018: A Systematic Review
A partir d'une revue systématique de publications d'essais cliniques ayant permis l'autorisation de 42 immunothérapies entre 2011 et 2018, cette étude analyse, à l'aide d'un système de score basé sur 24 critères issus des recommandations du consortium SISAQOL, la présence de résultats déclarés par les patients dans les rapports d'essais cliniques ainsi que la qualité de ces données
Résumé en anglais
Background : Patient-reported outcomes (PROs) promote patient-centeredness in clinical trials; however, in the field of the rapidly emerging and clinically impressive immunotherapy, data on PROs are limited.
Methods : We systematically identified all immunotherapy approvals from 2011 through 2018 and assessed the analytic tools and reporting quality of associated PRO reports. For randomized clinical trials (RCTs), we developed a novel 24-point scoring scale: the PRO Endpoints Analysis Score (PROEAS) based on 24 criteria derived from the recommendations of the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) Consortium.
Results : We assessed 44 trial publications supporting 42 immunotherapy approvals. PROs were published for 21 of the 44 (47.7%) trial publications. Twenty-three trials (52.3%) were RCTs and 21 (47.7%) pertained to single-arm trials. The median time between primary clinical outcomes publications and their corresponding secondary PRO publications was 19 months (IQR= 9–29). Of the 21 PRO reports, 4 (19.0%) reported a specific hypothesis, and most (85.7%) used descriptive statistics. Three (3 of 21 [14.3%]) studies performed a control for type I error. As for RCTs, 14 of 23 (60.9%) published PRO data including 13 (56.5%) that published a secondary dedicated manuscript. Half of these 14 trials scored < 13 points on the 24-point PROEAS. The mean score was 12.71 (range= 5–17; SD = 3.71), and none met all the recommendations of the SISAQOL Consortium.
Conclusion : Suboptimal reporting of PROs occurs regularly in cancer immunotherapy trials. Increased efforts are needed to maximize the value of this data in cancer immunotherapy development and approval.
