The ERICA Patient Reported Outcome Measures (PROMs) Repository is the first attempt to identify and centralize Clinical Assessment Outcomes questionnaires of relevance for rare diseases and constitutes a milestone in the Europe-wide standardization of Patient-Centered Outcome Measures (PCOMs) and PROMs for rare diseases.
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The COMET Initiative brings together people interested in the development and application of agreed standardised sets of outcomes, known as ‘core outcome sets’ (COS). These sets represent the minimum that should be measured and reported in all clinical trials of a specific condition, but COS are also suitable for use in routine care, clinical audit and research other than randomised trials
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Measuring health-related quality of life in patients with rare disease (Lenderking et al., 2021)
Published by ECRINArticle authors
Clinical Trials ToolkitThis article explores some of the challenges in HRQoL assessment in rare disease, propose solutions, and consider regulatory issues
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Patient reported outcome measures in rare diseases: a narrative review (Slade et al, 2018)
Published by ECRINArticle authors
Clinical Trials ToolkitThis review explores some of the current issues around the utilisation of PROMs in rare diseases, including small patient populations and dearth of valid PROMs. Difficulties in validating new or current PROMs for use in clinical trials and research are discussed
View this resource Bookmark this resourceDatabase of systematic reviews of outcome measurement instruments
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