OBJECTIVES: Clear cell renal cell carcinoma (ccRCC) is the most common type of renal cancer which is often diagnosed after tumours have spread from the kidneys. Given most patients undergoing treatment are suffering from metastatic disease, it is important to understand the wide range of symptoms and impacts and assess the validity of existing patient reported outcome measures (PROs) to adequately capture the burden of illness and benefit of treatment. This study evaluated symptoms and impacts of ccRCC and the validity of existing instruments.

METHODS: A targeted literature review was conducted to identify signs, symptoms, impacts and instruments. In parallel, searches of ClinicalTrials.gov, drug labels, and the Digital Medicine Society’s endpoint library were conducted to identify PROs relevant to patients with ccRCC. A gap analysis was conducted on a selection of the identified PROs to evaluate the development and psychometric properties against regulatory guidance. A preliminary conceptual model was developed which supported a mapping exercise of concepts to the shortlisted PROs.

RESULTS: N = 15 PRO measures were identified from searches; n = 11 were included in the gap analysis, of which n = 3 were kidney cancer specific (FKSI-15, FKSI-DRS and NFKSI-19). No digital endpoints were identified. None of the PROs met full criteria for content and psychometric validation in ccRCC as outlined by regulatory guidance. The NFKSI-19, as well as the general oncology measures MDASI and EORTC-QLQ-C30 had the most comprehensive coverage when compared to the conceptual model.

CONCLUSIONS: This study identified potential PROs for use in ccRCC studies. Further validation of these measures within the specific context of use is needed. As therapeutic innovation evolves, further research is also needed to capture patients’ experience of not only the symptoms and impacts of ccRCC but also the impacts of different treatments to ensure the instruments remain fit-for-purpose.