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Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?

Overview of attention for article published in Orphanet Journal of Rare Diseases, January 2017
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  • 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 (94th percentile)

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5 news outlets
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13 X users
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1 Facebook page

Citations

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75 Dimensions

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167 Mendeley
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Title
Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?
Published in
Orphanet Journal of Rare Diseases, January 2017
DOI 10.1186/s13023-016-0551-7
Pubmed ID
Authors

R. Rodriguez-Monguio, T. Spargo, E. Seoane-Vazquez

Abstract

More than 6,800 rare diseases and conditions have been identified in the US, which affect 25-30 million Americans. In 1983, the US Congress enacted the Orphan Drug Act (ODA) to encourage the development and marketing of drugs to treat rare diseases and conditions. This study analyzed all orphan designations and FDA approvals since 1983 through 2015, discussed the effectiveness of incentives for the development of treatments for rare diseases, and reflected on the ethical imperatives for timely access to orphan drugs. Study data were derived from the Food and Drug Administration (FDA) Orange Book and the Office of Orphan Drugs Development. A search was conducted to assess literature on the ethical principles and economic incentives for the development of orphan drugs. In the period 1983-2015, the FDA granted 3,647 orphan drug designations and 554 orphan drug approvals. The orphan drug approvals corresponded to 438 different brand names. Cancer was the therapeutic area with the highest number of approvals. The increased number of patients with rare diseases and the growth in the cost of orphan drugs pose a significant economic burden for patients, public programs and private third party payers. Regulatory differences to qualify for orphan designation and various population thresholds employed by the FDA and the European Medicines Agency lead to further unmet health needs for patients with rare diseases and aggravate health inequities. There is no societal consensus on the population and economic thresholds, the drug effectiveness indicator(s), or the societal value to be placed for the approval and reimbursement of orphan drugs. Orphan drug development and marketing in the US concentrate in few therapeutic areas. Despite the increase in the number of FDA approved orphan drugs, the unmet needs of patients with rare diseases evidence that the current incentives are not efficiently stimulating orphan drug development. There is need to balance economic incentives to stimulate the development and marketing of orphan drugs without jeopardizing patients' access to treatment. Thus, aligning pharmaceutical companies' incentives with societal budgetary constraints is necessary and the ethical imperatives of timely access to orphan drugs need to be agreed upon.

X Demographics

X Demographics

The data shown below were collected from the profiles of 13 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 167 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 167 100%

Demographic breakdown

Readers by professional status Count As %
Student > Master 35 21%
Researcher 22 13%
Student > Ph. D. Student 19 11%
Other 13 8%
Student > Bachelor 13 8%
Other 24 14%
Unknown 41 25%
Readers by discipline Count As %
Medicine and Dentistry 31 19%
Pharmacology, Toxicology and Pharmaceutical Science 21 13%
Social Sciences 18 11%
Nursing and Health Professions 12 7%
Biochemistry, Genetics and Molecular Biology 9 5%
Other 27 16%
Unknown 49 29%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 49. 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 20 October 2017.
All research outputs
#736,344
of 22,925,760 outputs
Outputs from Orphanet Journal of Rare Diseases
#60
of 2,633 outputs
Outputs of similar age
#17,508
of 420,807 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#3
of 52 outputs
Altmetric has tracked 22,925,760 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 96th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 2,633 research outputs from this source. They typically receive more attention than average, with a mean Attention Score of 7.6. 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 420,807 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 52 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 94% of its contemporaries.