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Table 5 PROM mentions: Concepts measured in each context of use

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

Measured quantity: Mention frequency

Number of articles published

HRQoL

Symptoms

Psychological and cognitive functioning

Physical functioning

Other

Total

Observational study/registry

49

34

15

13

8

17

87

Clinical trial

40

19

26

2

4

15

66

Questionnaire development/validation

20

9

6

1

5

11

32

Total

109

62

47

16

17

43

185