Exploring the Content and Psychometric Validity of Clinical Outcome Assessments in Pancreatic Ductal Adenocarcinoma Versus the Patient Reported Symptoms and Impacts

OBJECTIVES: Although pancreatic ductal adenocarcinoma (PDAC) is a leading cause of death in developed countries, treatment options remain scarce. In order to fully capture the potential benefit of new treatments, it is essential to understand the symptoms and impacts of the condition and the ability of existing patient reported outcome measures (PROs) to capture them robustly. This study evaluated the symptoms and impacts of PDAC 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 PDAC. A gap analysis was then conducted on a selection of the identified PROs to evaluate the development and psychometric properties against regulatory best practices. An existing conceptual model (Herman et al., 2019) was also extended which supported the mapping of concepts to the shortlisted PROs.

RESULTS: N = 19 PROs were identified from searches; n = 9 were selected for further examination in the gap analysis, of which n = 3 were pancreatic cancer specific (FACT-Hep, FACT-FHSI, and EORTC-QLQ-PAN26 combined with the EORTC-QLQ-C30). No digital endpoints were identified. None of the PROs met full criteria for content and psychometric validation in PDAC as outlined by regulatory guidance. The EORTC-QLQ-PAN26 combined with the EORTC-QLQ-C30 had the most comprehensive coverage when compared to the conceptual model.

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