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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 PROMsNumber of studies with a PROM measuring this concept% of total (out of 115 studies)
Symptoms3429.6
Health-related quality of life2219.1
Rescue medication108.7
Functioning (activities of daily living, disabilities)54.3
Health status, perceived general health43.5
Impact (e.g., of fatigue, disruptions at work/school)32.6
Global impression of change32.6
Compliance21.7
Measure of ease of administration of a device10.9
Disease activity10.9
  1. Source: Clinicaltrials.gov database search on July 1, 2017