1760P - Systematic review of quality of life (QoL) inclusion among endpoints, reporting and impact of QoL results in phase III non-inferiority trials of systemic treatments in oncology

Background

Quality of life (QoL) is an increasingly important endpoint in oncology clinical trials. In the non-inferiority (NI) setting, the experimental treatment should confirm to be preferable, and patient-reported outcomes should have a relevant role. Aim of this study was to assess the inclusion of QoL among endpoints in phase III NI trials, the presence of QoL results in the publications and the impact of QoL results on the interpretation of the study.

Methods

We performed a PubMed search for phase III NI trials published between 2012 and 2021, testing systemic treatments in adult patients with solid tumours. Trials were divided according to 4 NI strategies: (1) different treatments; (2) different route of administration of the same drug; (3) shorter duration of treatment; (4) omission of one or more drugs. Trials were analysed according to three endpoints: (1) proportion of publications including QoL among endpoints; (2) proportion of publications reporting QoL results; (3) proportion of publications with demonstration of NI, in which QoL results actually favour the experimental treatment.

Results

106 publications were eligible. In 47 studies (44.3%), QoL was not included among endpoints, and QoL results were not available in 66 (62.3%) primary publications. Out of 74 trials with demonstration of NI, QoL was not assessed in 36 (48.64%) and QoL results were not available in 43 primary publications (58.1%); QoL results actually favoured the experimental treatment in 19 trials (25.7%). Considering only the subgroup of 73 trials testing the NI of different treatments, 30 (41.1%) did not include QoL and QoL results were not available in 42 (57.5%) publications; within this subgroup, focusing on 55 trials with demonstration of NI, 25 (45.5%) did not include QoL; QoL results were not available in 29 (52.7%) primary publications; QoL results actually favoured the experimental treatment in 15 trials (27.3%).

Conclusions

Quality of life is not assessed and/or reported in many non-inferiority trials testing cancer treatments. Among trials formally demonstrating the non-inferiority, only a minority can claim quality of life results actually favouring the experimental treatment.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

L. Marandino: Financial Interests, Personal, Advisory Role: Gilead, Merck; Financial Interests, Personal, Research Grant: AstraZeneca; Non-Financial Interests, Personal, Other, Travel accommodation: Janssen. F. Perrone: Financial Interests, Personal, Advisory Board: AstraZeneca, Incyte, Ipsen, Astellas, Eli Lilly, GSK, Boehringer Ingelheim, Pfizer, BMS; Financial Interests, Institutional, Research Grant, Financial support for clinical trials promoted by the NCI Naples: Roche, Bayer, AstraZeneca, Pfizer, Merck; Financial Interests, Institutional, Research Grant, Financial support for phase I trials at the NCI Naples: Incyte, Tesaro/GSK; Non-Financial Interests, Leadership Role, I've been elected President and will be active from 2023 to 2025: AIOM Associazione Italiana di Oncologia Medica. M. Di Maio: Financial Interests, Personal, Advisory Board, Consultancy about clinical trial methodology and clinical trial results interpretation: Novartis; Financial Interests, Personal, Advisory Board, Consultancy about immunotherapy in SCLC: Roche; Financial Interests, Personal, Advisory Board, Consultancy about role and interpretation of patient-reported outcomes and quality of life in clinical trials: Takeda; Financial Interests, Personal, Advisory Board, Advisory board about the role of chemotherapy and hormonal treatment in hormone-sensitive prostate cancer: Janssen; Financial Interests, Personal, Advisory Board, Consultancy about the results obtained with lorlatinib and dacomitinib in advanced non-small cell lung cancer: Pfizer; Financial Interests, Personal, Advisory Board, Consultancy about role of osimertinib as adjuvant treatment of NSCLC: AstraZeneca; Financial Interests, Personal, Invited Speaker, Compensation for a talk and a document about role of patient-reported outcomes in clinical trials and in clinical practice, with a specific focus on lung cancer: Boehringer Ingelheim; Financial Interests, Personal, Advisory Board, Participation in advisory boards about olaparib in pancreatic cancer, about olaparib in prostate cancer, and about immunotherapy in lung cancer: Merck Sharp & Dohme; Financial Interests, Personal, Advisory Board, Consultant for the new indication of avelumab in urothelial cancer: Merck; Financial Interests, Institutional, Research Grant, Financial support and drug supply for the Meet-URO12 trial (niraparib as maintenance treatment of urothelial carcinoma after first-line treatment with platinum-based chemotherapy): Tesaro - GSK; Financial Interests, Institutional, Local PI, Local PI of trial with tislelizumab in hepatocellular carcinoma: BeiGene; Financial Interests, Institutional, Local PI, Local PI of a trial with cabozantinib and atezolizumab in advanced HCC: Exelixis; Financial Interests, Institutional, Local PI, Local PI of a trial with atezolizumab and bevacizumab in advanced HCC: Roche; Financial Interests, Institutional, Local PI, Local PI of trials with pembrolizumab in hepatocellular carcinoma: Merck Sharp & Dohme; Financial Interests, Institutional, Local PI, Local PI of a trial with sasanlimab in NMI bladder cancer: Pfizer. All other authors have declared no conflicts of interest.