EMA-EORTC workshop underscores critical role of patient-reported outcomes and quality of life data in regulatory decision-making

Highlights from a joint European Medicine Agency (EMA) and European Organisation for Research and Treatment of Cancer (EORTC) workshop.¹

Brussels, 15 April 2025 – A new article published yesterday by Pe, Voltz et al. in The Lancet Oncology² summarises highlights from a joint EMA-EORTC workshop held on 29 February 2024 in Amsterdam and aimed at clarifying the current use of patient-reported outcomes (PROs) and health-related quality of life (HRQOL) for evaluating anti-cancer treatments and facilitating interactions among relevant stakeholders to foster international collaboration. Workshop participants represented academia, learned societies, patients, regulatory agencies, health technology assessment bodies (HTA), and industry.

• PROs can support overall benefit-risk evaluation and further characterise tolerability;
• A thoughtful mix of validated HRQOL questionnaires and customised PRO measures (i.e., item lists) can help to ensure that PROs remain relevant and fit-for-purpose as treatment paradigms evolve;
• There is considerable overlap in the PROs that are considered relevant for different stakeholders, and it is crucial to consider what matters most to patients when selecting PROs;
• Several international multistakeholder initiatives are underway that support the need for robust methodology and adequate analysis and reporting of PROs within cancer clinical trials;
• PROs intended to provide quantitative assessment of clinical outcomes should be treated like any other endpoint included in the evaluation of an anti-cancer treatment, with clear research objectives and consistent methodological standards.

EORTC has always worked on improving the survival and HRQOL of patients through cancer research. For more than 40 years, the EORTC Quality of Life Group (QLG) academic network has dedicated its research activities to the development of reliable and valid PRO instruments that can be used in cancer clinical research. The EORTC QLG’s measurement strategy continues to evolve to fit the changing clinical trial landscape, ensuring a balance of static and flexible approaches to capture relevant patient outcomes.

EORTC also recognises the need for a set of agreed standards on how to design, analyse, and interpret PRO data in cancer clinical trials, and the importance of collaboration among international regulatory agencies, HTA bodies, industry, academia, and patient representative groups. Initiatives like Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints (SISAQOL) continue to address how PROs should be implemented to meet the needs of all stakeholders, ensuring the patient voice is incorporated in the decision-making process.

“While the benefit-risk assessment of anti-cancer treatments has historically focused on clinical outcomes such as overall survival, progression-free survival, tumour response, and clinician-reported adverse events, this workshop highlights that multiple stakeholders agree on the importance of incorporating PROs in order to measure symptoms, functioning, and other aspects of HRQOL from the patient perspective” said Dr Madeline Pe, co-lead author of this article. “This workshop underlines the shared interest of participants to continue discussions and collaborations aimed at supporting the inclusion of PROs within regulatory and HTA decision making” added Dr Jaap Reijneveld, Chair of the EORTC QLG.

The European Organisation for Research and Treatment of Cancer (EORTC) is a non-governmental, non-profit organisation, which unites clinical cancer research experts, throughout Europe, to define better treatments for cancer patients to prolong survival and improve quality of life. Spanning from translational to large, prospective, multi-centre, phase III clinical trials that evaluate new therapies and treatment strategies as well as patient quality of life, its activities are coordinated from EORTC Headquarters, a unique international clinical research infrastructure, based in Brussels, Belgium.

The EORTC Quality of Life Group (QLG) strives to improve health-related quality of life (HRQoL) of cancer patients, through dedicated research and the use of HRQoL measures within cancer clinical trials and clinical practice. HRQoL constitutes an important aspect of cancer research and care: it gives a voice to patients, putting their experience at the forefront. The QLG is part of the European Organisation for Research and Treatment of Cancer (EORTC).
For further information, please visit the QLG website: qol.eortc.org

Caroline Hance (EORTC QLG)
caroline.hance@eortc.org

¹ EMA and European Organisation for Research and Treatment of Cancer (EORTC) workshop: How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions? | European Medicines Agency (EMA)
² Madeline Pe et al. (2025). Using patient-reported outcomes and health-related quality of life data in regulatory decisions on cancer treatment: highlights from an EMA-EORTC workshop. The Lancet Oncology. https://doi.org/10.1016/S1470-2045(25)00150-0