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

TL;DR

This study evaluates the content validity of patient-reported outcome measures in neuro-oncology using the WHO ICF framework, finding that over half lack sufficient coverage.

Contribution

The study introduces a systematic method to assess PRO measures using the WHO ICF framework for neuro-oncology.

Findings

Only 37.8% of survey items were considered relevant by patients, proxies, or healthcare professionals.

Between 35% and 49% of PRO measures achieved sufficient content validity (≥80% coverage).

Multidimensional PRO measures showed insufficient content validity in more than half of cases.

Abstract

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.

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.

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).

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.

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