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Measuring what matters to rare disease patients – reflections on the work by the IRDiRC taskforce on patient-centered outcome measures

Overview of attention for article published in Orphanet Journal of Rare Diseases, November 2017
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About this Attention Score

  • In the top 5% of all research outputs scored by Altmetric
  • Among the highest-scoring outputs from this source (#44 of 2,123)
  • High Attention Score compared to outputs of the same age (94th percentile)
  • High Attention Score compared to outputs of the same age and source (90th percentile)

Mentioned by

twitter
67 tweeters
facebook
1 Facebook page
googleplus
1 Google+ user

Citations

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58 Dimensions

Readers on

mendeley
140 Mendeley
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Title
Measuring what matters to rare disease patients – reflections on the work by the IRDiRC taskforce on patient-centered outcome measures
Published in
Orphanet Journal of Rare Diseases, November 2017
DOI 10.1186/s13023-017-0718-x
Pubmed ID
Authors

Thomas Morel, Stefan J. Cano

Abstract

Our ability to evaluate outcomes which genuinely reflect patients' unmet needs, hopes and concerns is of pivotal importance. However, much current clinical research and practice falls short of this objective by selecting outcome measures which do not capture patient value to the fullest. In this Opinion, we discuss Patient-Centered Outcomes Measures (PCOMs), which have the potential to systematically incorporate patient perspectives to measure those outcomes that matter most to patients. We argue for greater multi-stakeholder collaboration to develop PCOMs, with rare disease patients and families at the center. Beyond advancing the science of patient input, PCOMs are powerful tools to translate care or observed treatment benefit into an 'interpretable' measure of patient benefit, and thereby help demonstrate clinical effectiveness. We propose mixed methods psychometric research as the best route to deliver fit-for-purpose PCOMs in rare diseases, as this methodology brings together qualitative and quantitative research methods in tandem with the explicit aim to efficiently utilise data from small samples. And, whether one opts to develop a brand-new PCOM or to select or adapt an existing outcome measure for use in a rare disease, the anchors remain the same: patients, their daily experience of the rare disease, their preferences, core concepts and values. Ultimately, existing value frameworks, registries, and outcomes-based contracts largely fall short of consistently measuring the full range of outcomes that matter to patients. We argue that greater use of PCOMs in rare diseases would enable a fast track to Patient-Centered Care.

Twitter Demographics

The data shown below were collected from the profiles of 67 tweeters who shared this research output. Click here to find out more about how the information was compiled.

Mendeley readers

The data shown below were compiled from readership statistics for 140 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 140 100%

Demographic breakdown

Readers by professional status Count As %
Researcher 36 26%
Student > Master 15 11%
Student > Ph. D. Student 13 9%
Other 9 6%
Student > Doctoral Student 9 6%
Other 28 20%
Unknown 30 21%
Readers by discipline Count As %
Medicine and Dentistry 36 26%
Social Sciences 13 9%
Biochemistry, Genetics and Molecular Biology 12 9%
Nursing and Health Professions 12 9%
Pharmacology, Toxicology and Pharmaceutical Science 6 4%
Other 28 20%
Unknown 33 24%

Attention Score in Context

This research output has an Altmetric Attention Score of 51. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 23 October 2021.
All research outputs
#576,366
of 19,467,557 outputs
Outputs from Orphanet Journal of Rare Diseases
#44
of 2,123 outputs
Outputs of similar age
#17,237
of 336,067 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#13
of 130 outputs
Altmetric has tracked 19,467,557 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 97th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 2,123 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 7.5. This one has done particularly well, scoring higher than 97% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 336,067 tracked outputs that were published within six weeks on either side of this one in any source. This one has done particularly well, scoring higher than 94% of its contemporaries.
We're also able to compare this research output to 130 others from the same source and published within six weeks on either side of this one. This one has done particularly well, scoring higher than 90% of its contemporaries.