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Table 4 Number of mentions and numbers of PROMs for therapeutic indications, concepts measured, PROM category, and PROM context of use

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

 N = MentionN = PROM
Therapeutic indications
 Hereditary angioedema133
 Hunter syndrome126
 Rare bone, joint and blood vessel disease99
 Systemic sclerosis86
 Pulmonary arterial hypertension76
 Adiposis dolorosa66
 Paroxysmal nocturnal hemoglobinuria66
 Ehlers Danlos hypermobile66
 Pompe disease65
Concepts measured by PROMs
 HRQoL6236
 Symptoms4731
 Physical functioning1715
 Psychological and cognitive functioning1615
 Other4326
PROM category
 Generic7227
 Non-rare disease specific4737
 Rare disease specific4734
 Study-specific1915
PROM context of use
 Observational study/registry8761
 Clinical trials6642
 Questionnaire development and validation3230