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Review of 11 national policies for rare diseases in the context of key patient needs

Overview of attention for article published in Orphanet Journal of Rare Diseases, March 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 (#24 of 2,029)
  • High Attention Score compared to outputs of the same age (96th percentile)
  • High Attention Score compared to outputs of the same age and source (99th percentile)

Mentioned by

news
4 news outlets
twitter
58 tweeters
facebook
5 Facebook pages

Citations

dimensions_citation
76 Dimensions

Readers on

mendeley
196 Mendeley
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Title
Review of 11 national policies for rare diseases in the context of key patient needs
Published in
Orphanet Journal of Rare Diseases, March 2017
DOI 10.1186/s13023-017-0618-0
Pubmed ID
Authors

Safiyya Dharssi, Durhane Wong-Rieger, Matthew Harold, Sharon Terry

Abstract

Rare diseases collectively exert a global public health burden in the severity of their manifestations and the total number of people they afflict. For many patients, considerable barriers exist in terms of access to appropriate care, delayed diagnosis and limited or non-existing treatment options. Motivated by these challenges, the rare disease patient community has played a critical role, elevating the patient voice and mobilizing legislation to support the development of programs that address the needs of patients with rare diseases.The US Orphan Drug Act of 1983 served as a key milestone in this journey, providing a roadmap for other countries to introduce and implement similar orphan drug legislation; more recently, the European Union (EU) has gone further to encourage the widespread adoption and implementation of rare disease plans or strategies designed to more adequately address the comprehensive needs of patients with rare diseases. Despite these legislative efforts and the growing contributions of patient advocacy groups in moving forward implementation and adoption of rare disease programs, gaps still exist across the policy landscape for several countries. To gain deeper insights into the challenges and opportunities to address key needs of rare disease patients, it is critical to define the current status of rare disease legislation and policy across a geographically and economically diverse selection of countries. We analyzed the rare disease policy landscape across 11 countries: Germany, France, the United Kingdom, Canada, Bulgaria, Turkey, Argentina, Mexico, Brazil, China, and Taiwan. The status and implementation of policy was evaluated for each country in the context of key patient needs across 5 dimensions: improving coordination of care, diagnostic resources, access to treatments, patient awareness and support, and promoting innovative research. Our findings highlight the continuing role of the patient community in driving the establishment and adoption of legislation and programs to improve rare disease care. Further, we found that while national rare disease plans provide important guidance for improving care, implementation of plans is uneven across countries. More research is needed to demonstrate the effect of specific elements of rare disease plans on patient outcomes.

Twitter Demographics

The data shown below were collected from the profiles of 58 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 196 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 196 100%

Demographic breakdown

Readers by professional status Count As %
Student > Master 36 18%
Researcher 32 16%
Student > Ph. D. Student 18 9%
Student > Bachelor 16 8%
Student > Doctoral Student 13 7%
Other 37 19%
Unknown 44 22%
Readers by discipline Count As %
Medicine and Dentistry 46 23%
Biochemistry, Genetics and Molecular Biology 27 14%
Pharmacology, Toxicology and Pharmaceutical Science 16 8%
Social Sciences 16 8%
Nursing and Health Professions 12 6%
Other 29 15%
Unknown 50 26%

Attention Score in Context

This research output has an Altmetric Attention Score of 76. 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 19 January 2021.
All research outputs
#368,152
of 18,949,038 outputs
Outputs from Orphanet Journal of Rare Diseases
#24
of 2,029 outputs
Outputs of similar age
#10,225
of 276,668 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#1
of 13 outputs
Altmetric has tracked 18,949,038 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 98th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 2,029 research outputs from this source. They typically receive more attention than average, with a mean Attention Score of 7.5. This one has done particularly well, scoring higher than 98% 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 276,668 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 96% of its contemporaries.
We're also able to compare this research output to 13 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 99% of its contemporaries.