Knowledge of the potential benefits and risks associated with the use of anticancer therapies is fundamental for making treatment-related recommendations and decisions. Two important oncology societies have recently taken a step forward to quantize the clinical benefit. The American Society of Clinical Oncology (ASCO) Value Framework (ASCO-VF), which was updated in 2016, and the European Society for Medical Oncology developed its Magnitude of Clinical Benefit Scale (ESMO-MCBS) for drugs indicated in the treatment of solid cancer, which also updated in 2017. They have been used to grade US Food and Drug Administration (FDA)-approved new drugs for treating advanced solid cancers. In the study by Vivot and colleagues, they found that Many recently FDA-approved new cancer drugs did not have high clinical benefit as measured by ASCO-VF and ESMO-MCBS.

The growing wave of progress using cancer immunotherapy, which has extended and improved the lives of patients, many of whom had few other effective treatment options has yielded high expectations from all stakeholders. However, there are also concerns about the value of check point inhibitors. Many immune checkpoint inhibitors were approved based on single-arm studies, only recently more RCTs were finished and reported.

Patient-reported outcomes (PROs), such as symptoms, quality of life (QOL), and patient-perceived health status supplement clinical data and are now more important during decision-making in oncology because they provide a holistic understanding of patient experience and treatment effectiveness. Both ESMO-MCBS and ASCO VF incorporated QOL into the determination of the value of a treatment. ASCO VF awarded bonus points for treatment with a statistically significant improvement in cancer-related symptoms. However, PROs usually were not reported in the primary report or approval documents, but subsequently reported as separate articles.

In this study, we aimed to describe the clinical benefit of checkpoint inhibitors that were recently approved by the FDA based on RCTs using ESMO-MCBS and ASCO VF, and whether these agents reach defined thresholds of long-term benefit in the two value frameworks. We also compare the values based on primary reports with those assessed based on updated reports including long-term survival reports and/or QOL reports.