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Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers

Overview of attention for article published in Orphanet Journal of Rare Diseases, May 2016
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About this Attention Score

  • In the top 25% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (91st percentile)
  • High Attention Score compared to outputs of the same age and source (96th percentile)

Mentioned by

blogs
2 blogs
twitter
13 tweeters
facebook
2 Facebook pages

Citations

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28 Dimensions

Readers on

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72 Mendeley
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Title
Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers
Published in
Orphanet Journal of Rare Diseases, May 2016
DOI 10.1186/s13023-016-0444-9
Pubmed ID
Authors

T. Morel, S. Aymé, D. Cassiman, S. Simoens, M. Morgan, M. Vandebroek

Abstract

Rare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context. A mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents. Final study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk. This is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations.

Twitter Demographics

The data shown below were collected from the profiles of 13 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 72 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 72 100%

Demographic breakdown

Readers by professional status Count As %
Student > Master 13 18%
Student > Ph. D. Student 11 15%
Researcher 9 13%
Student > Bachelor 7 10%
Other 6 8%
Other 15 21%
Unknown 11 15%
Readers by discipline Count As %
Medicine and Dentistry 17 24%
Pharmacology, Toxicology and Pharmaceutical Science 8 11%
Social Sciences 6 8%
Nursing and Health Professions 5 7%
Psychology 5 7%
Other 17 24%
Unknown 14 19%

Attention Score in Context

This research output has an Altmetric Attention Score of 23. 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 18 June 2018.
All research outputs
#1,023,096
of 17,361,274 outputs
Outputs from Orphanet Journal of Rare Diseases
#84
of 1,839 outputs
Outputs of similar age
#22,389
of 272,873 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#2
of 31 outputs
Altmetric has tracked 17,361,274 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 94th percentile: it's in the top 10% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 1,839 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 95% 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 272,873 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 91% of its contemporaries.
We're also able to compare this research output to 31 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 96% of its contemporaries.