Cancer is now diagnosed and treated earlier, resulting in improved patient benefit and outcomes. While overall survival (OS) is crucial to patients, there are other value dimensions, such as quality of life (QoL) and reduction in severe side effects, that change patient lives while on treatment. Considering patient reported outcomes (PROs) in value assessments for decision-making can improve individual, population and societal outcomes.

A multidisciplinary, international group of experts working in the cancer field was brought together to reach consensus on key principles of defining and assessing cancer treatment value. A Delphi-based approach including surveys, virtual panels, interviews and structured online discussions was used to reach consensus. This work was funded by AstraZeneca.

Use of PROs in oncology value assessment is important because it can lead to: improvements in caregiver/patient/physician communication; unmet problem and needs detection; disease and treatment tracking; and better cost effectiveness. While some health technology assessment (HTA) bodies are already accepting QoL data, such as the Institute for Quality and Efficiency in Health Care (IQWiG) in Germany and US Centers for Medicare and Medicaid (CMS), many others do not. It is important that there is consistency in use of QoL data and other PROs, ensuring inclusion and a standardized and simple way of capture. In trials, tolerability data collected via PROs and QoL should be routinely and consistently incorporated and emphasized in HTA value assessments together with safety, efficacy and effectiveness. Data from PROs should be considered in decision-making to help build a better picture of health-related QoL, morbidity and adverse events from the patient perspective.

We are calling on the cancer community to: continue to encourage the use of a broad set of oncology-relevant endpoints in clinical trials to further validate their relevance as endpoints either as predictors of clinical outcomes or endpoints with intrinsic value; collect complementary PRO data in clinical trials and as real-world evidence to better tailor treatment options to the outcomes that individual patients value most; involve patients in HTA processes.