Making patient experience count: the role of patient-reported outcomes in cancer trials

At the Cancer Drug Development Forum (CDDF) Workshop 2026, held on March 30, 2026, in Brussels, Belgium, our Executive Director, Alex Filicevas, spoke during the session “Challenges in the Landscape of a Global Development Strategy, including patient access”, focusing on the role of patient-reported outcomes (PROs) in cancer clinical trials.

PROs are used in clinical trials to capture how patients feel during and after treatment. They provide important insight into symptoms, side effects and quality of life: areas that are not always reflected in clinical data alone. During the discussion, Alex highlighted a key issue: while PROs are increasingly included in trials, they are often not designed in a way that allows them to influence regulatory or treatment decisions.

He explained that PROs are frequently added too late in the trial process, often as secondary endpoints, without a clear purpose. As a result, the data collected may be difficult to interpret and is rarely used by regulators or health authorities. The discussion also highlighted a broader gap between what is measured in trials and what patients actually experience. Patients consistently report that aspects such as quality of life, treatment burden and daily functioning matter most, yet these are not always captured in trial design.

Key messages from the session included:

• PROs need to be designed from the beginning of a trial, not added later
• Patient experience should be treated as decision-relevant evidence
• Condition-specific PROs are essential to capture what truly matters to patients
• Data that is not designed for decision-making is unlikely to influence access to treatments

In bladder cancer, PROs can help better understand issues such as urinary symptoms, sexual health and psychological impact, factors that are important for patients when making treatment decisions but are not always reflected in clinical trial outcomes.

Alex also emphasised the role of patient organisations in shaping more meaningful research. By bringing forward evidence from patient communities and contributing to trial design early on, they can help ensure that research reflects real-world patient needs.

Overall, the session reinforced a clear message: patient experience should be built into clinical trials from the start to ensure it meaningfully informs decisions and improves care for people affected by cancer.

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