Establishing the content validity of patient-reported outcome measures used in neuro-oncology based on the WHO ICF framework: Part of the RANO-PRO initiative
Year: 2025
Type: article
Study design: Observational Study
Abstract: BACKGROUND: Instruments to assess patient-reported outcomes (PRO) should generate high-quality evidence. Reliable PRO evidence is essential to policymakers, in conjunction with outcomes such as survival and radiological response, to understand the net clinical benefit of antitumor treatments. This study aimed to establish the content validity of 215 identified PRO measures used in patients with brain tumors. METHODS: A survey (n = 148 items) was developed reflecting aspects of the WHO International Classification of Functioning, Disability, and Health (ICF) framework. Patients with brain tumors, their proxies, and healthcare professionals (HCPs) were asked to rate each survey item on relevance. An item was considered a relevant issue if ≥25% of the patients or proxies or ≥50% of the HCPs considered that item to be an issue. Next, all items in the identified PRO measures were linked to ICF and relevant items in the survey, and the percentage of content coverage was calculated. RESULTS: In total, 114 patients, 71 proxies, and 65 HCPs from different countries completed the survey. Fifty-six of 148 (37.8%) items in the survey were considered relevant. The most important aspects mentioned by both patients and proxies were difficulty concentrating, difficulty remembering, multitasking, and handling stress. Depending on the definition, between 35% and 49% of PRO measures were considered to have sufficient content validity (≥80% coverage). CONCLUSION: The content validity was insufficient in more than half of the identified PRO measures, particularly multidimensional measures. Future research should investigate whether different approaches to PRO assessment better meet the needs of all stakeholders.
Source: Neuro-Oncology
Authors: Ogechukwu A Asogwa, Linda Dirven, Tobias Walbert, Terri S Armstrong, David Arons, Martin J van den Bent, Jaishri Blakeley, Marijke B Coomans, Paul D Brown, Helen Bulbeck, Susan M Chang, Corneel Coens, Mark R Gilbert, Robin Grant, Rakesh Jalali, Johan A F Koekkoek, Pankaj Kumar Panda, Danielle Leach, Heather Leeper, Tito Mendoza, Lakshmi Nayak, Kathy Oliver, Jaap C Reijneveld, Emilie Le Rhun, Larry Rubinstein, Jennie W Taylor, Michael Weller, Patrick Y Wen, Martin J B Taphoorn
Institutions: Leiden University Medical Center, Henry Ford Health, Wayne State University, Michigan State University, National Institutes of Health, National Cancer Institute, Center for Cancer Research, National Brain Tumor Society, Erasmus MC Cancer Institute, Johns Hopkins University, Johns Hopkins Medicine, Mayo Clinic, University of California, San Francisco, European Organisation for Research and Treatment of Cancer, Edinburgh Royal Infirmary, Apollo Proton Cancer Centre, University of Chicago, Center for Neuro-Oncology, Dana-Farber Cancer Institute, Amsterdam University Medical Centers, Stichting Epilepsie Instellingen Nederland, University of Zurich, University Hospital Zurich
Language: English
Topic: Cancer survivorship and care
Subfield: Oncology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $5,132
Funders: Pfizer, GlaxoSmithKline, Sontag Foundation, American College of Radiology Imaging Network, Wayne State University, Henry Ford Health System, Michigan State University, Johns Hopkins University, Regeneron Pharmaceuticals, European Association of Neuro-Oncology, Jazz Pharmaceuticals, Novocure, GW Pharmaceuticals, Incyte, ECOG-ACRIN Cancer Research Group, Amsterdam University Medical Centers, Seagen, Universiteit Leiden, ZonMw, Leids Universitair Medisch Centrum, Universität Zürich, European Society for Medical Oncology, Servier, Medac, Stichting Epilepsie Instellingen Nederland, National Institutes of Health, College of Engineering, Michigan State University, University of California, San Francisco, National Cancer Institute, Daiichi Sankyo Europe
OpenAlex ID: W4409870294