Developing a strategy to ensure EORTC QLG measures fit changing standards for cancer clinical trials

Health-related quality of life (HRQOL) and patient-reported outcomes (PROs) are gaining importance in cancer clinical trials, with regulatory bodies like FDA and EMA encouraging their inclusion in drug development. EMA and EORTC jointly organized a workshop on February 29, 2024, focusing on how PROs and HRQoL data can inform regulatory decisions, and FDA issued final guidelines on core PROs in cancer trials (October 2024), recommending collecting and analysing disease-related symptomatic adverse events, overall side effect impact summary measure, physical function, and role function. This shift in regulatory thinking motivates sponsors to prioritize PROs, and widely used tools like EORTC QLG measures must meet evolving standards. To ensure these measures satisfy the criteria of a good quality measure, are up to date with ever changing standards, and address the current needs of regulatory bodies, the EORTC QLG aims to: (1) develop a framework meeting regulatory and HTA criteria, (2) provide published evidence supporting the quality of its measures, using the QLQ-C30 as a case study, and (3) create a sustainability plan, including a searchable database of evidence. Reliable PRO measures help assess both treatment effectiveness and patient well-being in cancer care.

The project started in October 2024. The protocol for the scoping review has been developed and published. A search strategy has been developed for all bibliographic databases and grey literature has been identified. Screening of titles and abstracts and full-text screening has been completed. Data extraction is underway.

An a priori framework for the development and validation of a PROM has been developed. This framework will be used to map the steps described in the guidance documents retrieved in the literature review.

Complete the scoping review within 6 months. Subsequently plan consensus discussions with QLG expert panel and external stakeholders.

Agree on a final framework that meets the various quality criteria for a good PROM and subsequent discussions with various stakeholders in the next QLG meeting in 2026.

Patient-reported outcomes (PROs), like quality of life and symptom tracking, are becoming more important in cancer clinical trials. Regulatory agencies, like the FDA and EMA, now encourage pharma companies to include PROs when testing new cancer treatments.  EMA and EORTC held a workshop (on February 29, 2024) on how PROs can help make decisions about new medicines, and in October 2024, FDA issued guidelines recommending the collection of key PROs. This shift means pharma companies are more likely to consider patient experiences when developing new treatments. Since EORTC quality-of-life tools are widely used, they must stay up to date with evolving standards. The EORTC QLG plan includes creating a framework that meets regulatory and HTA requirements, publishing evidence on the reliability of their measures (using QLQ-C30 as a case study), and developing a searchable database of research.  Reliable PRO measures help ensure that cancer treatments not only extend life but also improve patients’ well-being.