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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Use of Biomarkers in Orphan Drug Development
Published by IRDiRC
Biomarker DevelopmentResearch and Drug DevelopmentRegulatory AffairsClinical DevelopmentEndpoint BiomarkerOrphan DrugEndpointBiomarkers can be utilized as primary surrogate endpoint as a basis for accelerated approval (US), and as secondary or exploratory endpoints for drug mechanism of action or aspects of Safety monitoring.
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Patient surveys / Patient Preferences studies / Ethnographic research
Published by IRDiRC
Endpoint PatientResearch and Drug DevelopmentClinical DevelopmentQualitative AssessmentPatient Preference StudyEthnographic ResearchQualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions.
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Development and use of Patient-Centered Outcome Measures
Published by IRDiRC
Research and Drug DevelopmentClinical DevelopmentEndpointBiomarkerPatient-Centred Outcome MeasurePatient-Centred Outcomes Measures (PCOMs) are questionnaires that ‘directly’ quantify the impact of a disease and treatment on health outcomes that matter to patients (as identified or affirmed by patients themselves, or their caregivers)
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Feasibility-Patient engagement in trial endpoint selection
Published by IRDiRC
Clinical TrialOutcome MeasureTrail Endpoint SelectionResearch and Drug DevelopmentClinical DevelopmentEndpointPatient EngagementGuidance on the pivotal clinical trials outcomes measures .The important of the patient’s feedback. Academics can then assess whether there is an outcome measure available that can measure this – if not, outcome measure(s) may need to be developed.
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