Table 3 PROM categories, definitions, and examples
PROM category name | Definition | Example(s) |
|---|---|---|
Rare disease-specific | Developed for a particular rare disease and used in a trial for that disease | Hemophilia-Specific Quality of Life Index for Children Questionnaire used in a trial for children with hemophilia |
Non-rare disease- specific | Developed for a disease which is not a rare disease, but used in a rare disease trial | Dermatology Life Quality Index used in trials for adult patients with pemphigus vulgaris or facial angiofibroma |
Generic | Developed for generic use and used in a rare disease trial | SF-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-specific | A measure whose name is not provided or is defined as study-specific | Quality of life changes over time and between the treatment groups used in trials with patients with immune thrombocytopenic purpura |