Background: Non-small cell lung cancer (NSCLC) accounts for 80% of lung cancers in the US, with a prevalence of 200,000 US adults in 2020. The treatment landscape is rapidly evolving with the emergence of novel treatments and combinations. Patient-reported outcome (PRO) data, an important resource in the field of oncology, has the potential to offer direct insight into the patient’s treatment experience, beyond efficacy and safety measures. Methods: A targeted literature search for PROs was performed using ePROVIDE, an online resource of clinical outcomes assessment, for inclusion of PRO claims in FDA or EMA drug labeling, and clinicaltrials.gov for PRO inclusion as an endpoint in NSCLC randomized clinical trials. Prescribing information was reviewed from ePROVIDE, and PRO-related labeling language and endpoints were extracted. In clinicaltrials.gov, an advanced search was conducted for NSCLC with the following parameters: study status, study phase 2 or 3, age greater than 18, and time frame within 10 years. Search terms used were “patient-reported outcome”, “HRQOL”, “time to deterioration”, “physical functioning”, and “pain”. Results: Search of ePROVIDE resulted in 17 approved medications, 14 of which were by the EMA, and 3 by the FDA. Among these labels, six types of PRO endpoints were identified: overall global quality of life, health-related quality of life, symptoms, health status, and pain. These endpoints were measured by several validated instruments, including the LCSS, EQ-5D, FACT-L, TOI, EORTC QLQ-C30, and EORTC QLQ-LC-13. The search of clinicaltrials.gov yielded 67 studies. Of these, 18 clinical trials had results. The other 50 trials were ongoing, pending results. The findings are summarized in Table 1. Endpoints were measured by 10 validated PRO instruments, listed in order of frequency: EORTC QLQ-C30, EORTC QLQ-LC-13, LCSS, EQ-5D, FACT-L, EQ-5D, LCSS, FACT-L, PRO-CTCAE, SF-15, CTSQ-16, BDI-II, and LASA. Only 2 clinical trials were mentioned in the respective prescribing information of the medications used as the intervention. Conclusion: To date, PRO endpoints were more likely to be included in EU prescribing information than US, and inclusion of patient-reported information could be valuable to patients, prescribers, and payer. Based on this database review, symptoms, physical functioning, and overall quality of life are an integral part of NSCLC clinical trials, emphasizing the importance of PRO endpoints in this patient population.
ClinicalTrials.Gov Search Results