Analysis of patient reported outcomes included in the registrational clinical trials of nivolumab for advanced non-small cell lung cancer

In the era of value-based oncology care, stakeholders are increasingly using patient reported outcomes (PROs) to guide clinical and regulatory decisions. PROs are also included in health technology assessments to guide patient access, drug reimbursement and pricing. We reviewed PROs collected in the United States Food and Drug Administration approved indications of nivolumab in advanced NSCLC. We analyzed the PRO data reported in the CheckMate 9LA (NCT03215706), CheckMate 227 (NCT02477826), CheckMate 057 (NCT01673867), and CheckMate 017 (NCT01642004) registrational clinical trials, and concluded that nivolumab alleviated symptom burden and improved health status of patients in this setting. However, inability of the included PRO instruments to measure immune-related adverse events, differences in the timing of PRO evaluation between treatment groups, incomplete patient participation at all time points, limited patient participation in the later time points, and interpretation of the longitudinal data are key challenges that impede accurate analysis and validation of PROs.