Outcome measurement in neurodegenerative disease: attributes, applications & interpretation

Neurodegener Dis Manag. 2015 Aug;5(4):305-16. doi: 10.2217/nmt.15.25. Epub 2015 Aug 21.

Abstract

There has been a marked shift from clinically assessed to patient assessed outcomes of treatment in neurodegenerative conditions over recent decades. The use of patient-reported outcome measures (PROMs) provides a method with which researchers and clinicians can gain insightful and meaningful data on health status from the patients' perspective. It is imperative that high-quality PROMs are chosen based upon their measurement properties and their suitability for use in the intended clinical or research context. This review aims to give a brief overview of best practice standards for selecting PROMs, current instruments used in exemplar neurodegenerative conditions and their application in clinical trials and routine measurement.

Keywords: clinical trial; disease specific; generic; neurodegenerative outcomes; patient-reported outcome measure; questionnaire; reliability; responsiveness; validity.

Publication types

  • Review

MeSH terms

  • Health Status
  • Humans
  • Neurodegenerative Diseases / diagnosis*
  • Patient Outcome Assessment*
  • Quality of Life
  • Reproducibility of Results
  • Sensitivity and Specificity
  • Surveys and Questionnaires