Reporting of patient reported outcomes in randomized clinical trials (RCTs) in non-small cell lung cancer (NSCLC) patients (pts).

Abstract

6573 Background: A patient-centric approach to clinical trials has become necessary, with regulators emphasizing the importance of pts' experience. PRO is of utmost relevance for pts with cancers such as NSCLC, that are likely to be highly symptomatic and/or with an unfavorable prognosis. Methods: To evaluate the rate at which PRO included in clinical trials are made publicly available when trial results are published, we searched Citeline Trialtrove, a registry of clinical trials, for NSCLC phase 3 RCTs, recruiting during 2017-2022, with PRO listed - regardless of publication type. We excluded supportive care, radiotherapy, and unpublished trials. We identified publications associated with the trial, and extracted various parameters, including if PRO outcome was published. Results: Of 122 NSCLC RCTs identified, 66 listed at least 1 PRO. Trials were reported as full-text (43), abstract (9), 1 only in a trials registry; 13 not published. Fifty-three trials were included, all had PRO as a secondary endpoint. The sponsor was industry in the majority of the trials. Most included stage III/IV NSCLC pts, and 1st/2nd line or maintenance. 24 PRO tools were used, mainly EORTC QLQ LC13 (35 trials), EORTC QLQ C30 (34), EQ-5D-5L (14), LCSS (8), EQ-5D-3L (6). Seven trials listed 1 tool, 26 listed 2, 18 listed >3. PRO was reported in 36 (68%) of the published trials. In the subgroup of trials with disease control (DC) benefit and no overall survival (OS) benefit, PRO was reported in 15/21 (71%) trials. PRO benefit was shown in 9 of them. Of 43/53 trials published as full-text, 31 (72%) had PRO reported. A benefit in at least 1 PRO tool was reported in 21 studies, 9 did not show a PRO benefit; in 5 trials, only descriptive and no comparative analysis of PRO was reported. Of the 21 trials with PRO benefit, 19 had a DC benefit and 10 had also OS benefit. PRO was reported together with the first reporting of other outcomes in 18 (50%) trials, and after 7-32 months after the initial one in 18 trials. Conclusions: Of 122 RCTs only 66 had at least 1 planned PRO, and only 35 had it published. Even where full-text was available or no OS benefit shown, about 1/3 of the trials did not report PRO. Characteristics as disease stage, therapy line, sponsor, and primary endpoint did not differ between all included trials and those reporting PRO. The reporting rate increased compared to our previous report yet it is not satisfactory. The importance of quality of life and pts centric outcomes, and the great efforts needed from pts and operators to collect the data, should translate into routine, timely and appropriate reporting of PRO to allow a thorough assessment of treatment effect. [Table: see text]