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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 = 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