PROMs Repository

The ERICA Patient-Reported Outcome Measures (PROMs) Repository is a centralised resource designed to facilitate the identification and use of patient-centred outcome measures in rare disease research. It provides access to information on available PROMs, observer-reported outcome measures (ObsROs) and other patient-centred clinical outcome assessment (PC-COA) tools, supporting researchers in selecting appropriate instruments for clinical studies and clinical trial protocols.

During protocol development, the repository can help researchers identify existing measurement instruments that may be suitable for assessing outcomes meaningful to patients, including symptoms, functioning, quality of life, daily activities and treatment impact. The resource allows users to explore measures according to relevant characteristics, such as target disease, age group, outcome type and rare disease area, supporting informed decisions when defining endpoints and selecting assessment tools.

The repository is particularly valuable for rare disease clinical trials, where outcome measurement can be challenging due to small patient populations, disease heterogeneity and limited availability of validated disease-specific instruments. By improving access to existing PROMs and patient-centred outcome measures, it helps researchers avoid unnecessary duplication, identify potentially suitable instruments, and strengthen the relevance and interpretability of trial outcomes.

As part of the ERICA patient-centred research activities, the repository also supports the integration of patient perspectives into outcome assessment by promoting the adoption of standardised measures developed or validated with input from rare disease communities. It complements methodological guidance on PROM selection and validation by providing a practical starting point for researchers designing patient-centred rare disease clinical trial protocols.