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Table 6 PROMs: categories used 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: PROM frequency

Number of articles published

Generic

Non-rare disease

Rare disease specific

Study-specific

Total PROMs

Observational study/registry

49

19

26

9

7

61

Clinical trials

40

12

13

10

7

42

Questionnaire development/validation

20

7

3

18

2

30

Total

109

38

42

37

15

132

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172