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Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints

Overview of attention for article published in Orphanet Journal of Rare Diseases, February 2015
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About this Attention Score

  • In the top 5% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (95th percentile)
  • High Attention Score compared to outputs of the same age and source (97th percentile)

Mentioned by

news
1 news outlet
blogs
2 blogs
twitter
18 X users
facebook
5 Facebook pages

Citations

dimensions_citation
47 Dimensions

Readers on

mendeley
119 Mendeley
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Title
Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints
Published in
Orphanet Journal of Rare Diseases, February 2015
DOI 10.1186/s13023-014-0195-4
Pubmed ID
Authors

Emil D Kakkis, Mary O’Donovan, Gerald Cox, Mark Hayes, Federico Goodsaid, PK Tandon, Pat Furlong, Susan Boynton, Mladen Bozic, May Orfali, Mark Thornton

Abstract

For rare serious and life-threatening disorders, there is a tremendous challenge of transforming scientific discoveries into new drug treatments. This challenge has been recognized by all stakeholders who endorse the need for flexibility in the regulatory review process for novel therapeutics to treat rare diseases. In the United States, the best expression of this flexibility was the creation of the Accelerated Approval (AA) pathway. The AA pathway is critically important for the development of treatments for diseases with high unmet medical need and has been used extensively for drugs used to treat cancer and infectious diseases like HIV.

X Demographics

X Demographics

The data shown below were collected from the profiles of 18 X users who shared this research output. Click here to find out more about how the information was compiled.
Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 119 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
United States 2 2%
Netherlands 1 <1%
Belgium 1 <1%
Unknown 115 97%

Demographic breakdown

Readers by professional status Count As %
Student > Ph. D. Student 19 16%
Researcher 17 14%
Other 13 11%
Student > Master 9 8%
Professor 6 5%
Other 28 24%
Unknown 27 23%
Readers by discipline Count As %
Medicine and Dentistry 26 22%
Biochemistry, Genetics and Molecular Biology 9 8%
Nursing and Health Professions 9 8%
Social Sciences 9 8%
Agricultural and Biological Sciences 6 5%
Other 19 16%
Unknown 41 34%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 33. 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 07 July 2021.
All research outputs
#1,195,313
of 25,374,917 outputs
Outputs from Orphanet Journal of Rare Diseases
#120
of 3,105 outputs
Outputs of similar age
#16,600
of 366,748 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#1
of 36 outputs
Altmetric has tracked 25,374,917 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 95th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 3,105 research outputs from this source. They typically receive more attention than average, with a mean Attention Score of 8.2. This one has done particularly well, scoring higher than 96% 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 366,748 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 95% of its contemporaries.
We're also able to compare this research output to 36 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 97% of its contemporaries.