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Table 3 PROM categories, definitions, and examples

From: To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases

PROM category nameDefinitionExample(s)
Rare disease-specificDeveloped for a particular rare disease and used in a trial for that diseaseHemophilia-Specific Quality of Life Index for Children Questionnaire used in a trial for children with hemophilia
Non-rare disease- specificDeveloped for a disease which is not a rare disease, but used in a rare disease trialDermatology Life Quality Index used in trials for adult patients with pemphigus vulgaris or facial angiofibroma
GenericDeveloped for generic use and used in a rare disease trialSF-36 used in trials for adults with Addison’s disease or EQ-5D used in trials for adults with progressive supra-nuclear palsy
Includes generic measures used in populations of children and the elderly
PedsQL used in trials for children with mitochondrial diseases, cystinosis, or childhood idiopathic nephrotic syndrome
Study-specificA measure whose name is not provided or is defined as study-specificQuality of life changes over time and between the treatment groups used in trials with patients with immune thrombocytopenic purpura