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Table 1 Concepts measured by PROMs included in rare disease studies

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

Concept assessed by PROMs

Number of studies with a PROM measuring this concept

% of total (out of 115 studies)

Symptoms

34

29.6

Health-related quality of life

22

19.1

Rescue medication

10

8.7

Functioning (activities of daily living, disabilities)

5

4.3

Health status, perceived general health

4

3.5

Impact (e.g., of fatigue, disruptions at work/school)

3

2.6

Global impression of change

3

2.6

Compliance

2

1.7

Measure of ease of administration of a device

1

0.9

Disease activity

1

0.9

  1. Source: Clinicaltrials.gov database search on July 1, 2017