Outcome measurement in neurodegenerative disease: attributes, applications & interpretation

Written by Laura Kelly, Crispin Jenkinson & David Morley

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.

Practice points

  • Patient-reported outcome measures are now central to the evaluation of healthcare.

  • After considering the suitability of an instrument in relation to the construct of interest, potential users should consider the fundamental measurement properties of an instrument, that is, reliability, validity and responsiveness, prior to selection. Such properties are widely endorsed by regulatory bodies.

  • Measures can be particular to a specific condition (i.e., PDQ-39; MSIS-29) or generic in nature and thereby used across a range of conditions (i.e., SF-36; EQ-5D).

  • Patient-reported outcome measures are increasingly incorporated into clinical trials, giving important insight into the impact of new treatments on patients’ self-reported functioning and well-being.

  • Future developments are likely to include exploring the use of measures at an individual level, electronic data capture and computer adaptive testing.

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