Health-related quality of life measurement strategy for solid rare cancers

Patients diagnosed with a rare tumour type report poorer psychosocial outcomes and impaired health-quality of life (HRQoL). HRQoL is often assessed with generic measures lacking content validity. Specific HRQoL assessment is important to understand the variances in quality of life patients experience. This study aims to examine how HRQoL is currently assessed in clinical solid rare cancer research and identify specific HRQoL issues patients experience. Moreover, we aim to identify specific issues patients with a solid rare cancer experience in the healthcare system related to the rarity of their disease, and test the content validity of the stand-alone healthcare-related and tumour-specific EORTC questionnaires for patients with a solid rare cancer at the same tumour-location. We will conduct an international, multicentre, mixed-methods study among solid rare cancer patients >18 years. First, systematic literature reviews will be performed. Subsequently, we will perform semi-structured interviews with patients and healthcare professionals (HCPs) and ask them to rate the EORTC questionnaires for their particular (rare) cancer experience. Basic quantitative analyses, including descriptive statistics (e.g. % missing data, mean and standard deviations) will be used to analyze patient and HCPs data. The outcomes of the project will result in standards of the HRQoL measurement in patients diagnosed with a solid rare cancer. 

We have received ethical approval at more than 30 collaborating centres and started the local data collection. 22 countries are involved in the project, worldwide. The first literature review is currently been written and will be published at the end of 2024.   

Current status:

Recruitment of patients and healthcare professionals.  

Continue with data collection until end of 2024. We hope that by Spring 2025 all participant data will be completed.    

If you are interested in collaborating in the project, please send an email to c.simoes@nki.nl

Rare cancers consist of more than 186 types. We know that the type, location, and stage of cancer, the type of treatment, as well as sociodemographic variables such as age and sex can be associated with the level of health-related quality of life (HRQoL) of patients. Because of its heterogeneity, it is fundamental to assess the impact of the diagnosis and treatment in terms of HRQoL. However, most time these assessments use generic questionnaires, lacking specific instruments. It is important to understand these variances when establishing a HRQoL measurement strategy. This study aims to raise standards of HRQoL measurement in patients diagnosed with a rare solid tumour type. Such a measurement strategy will ensure outcomes that matter most to rare cancer patients are encapsulated in future research and clinical practice.