| N = Mention | N = PROM |
---|---|---|
Therapeutic indications | ||
 Hereditary angioedema | 13 | 3 |
 Hunter syndrome | 12 | 6 |
 Rare bone, joint and blood vessel disease | 9 | 9 |
 Systemic sclerosis | 8 | 6 |
 Pulmonary arterial hypertension | 7 | 6 |
 Adiposis dolorosa | 6 | 6 |
 Paroxysmal nocturnal hemoglobinuria | 6 | 6 |
 Ehlers Danlos hypermobile | 6 | 6 |
 Pompe disease | 6 | 5 |
Concepts measured by PROMs | ||
 HRQoL | 62 | 36 |
 Symptoms | 47 | 31 |
 Physical functioning | 17 | 15 |
 Psychological and cognitive functioning | 16 | 15 |
 Other | 43 | 26 |
PROM category | ||
 Generic | 72 | 27 |
 Non-rare disease specific | 47 | 37 |
 Rare disease specific | 47 | 34 |
 Study-specific | 19 | 15 |
PROM context of use | ||
 Observational study/registry | 87 | 61 |
 Clinical trials | 66 | 42 |
 Questionnaire development and validation | 32 | 30 |