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Sustainable rare diseases business and drug access: no time for misconceptions

Overview of attention for article published in Orphanet Journal of Rare Diseases, July 2013
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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 (89th percentile)
  • High Attention Score compared to outputs of the same age and source (93rd percentile)

Mentioned by

policy
3 policy sources
twitter
7 X users
facebook
1 Facebook page

Citations

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28 Dimensions

Readers on

mendeley
150 Mendeley
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Title
Sustainable rare diseases business and drug access: no time for misconceptions
Published in
Orphanet Journal of Rare Diseases, July 2013
DOI 10.1186/1750-1172-8-109
Pubmed ID
Authors

Pierrick Rollet, Adrien Lemoine, Marc Dunoyer

Abstract

Legislative incentives enacted in Europe through the Regulation (EC) No. 141/2000 to incentivize orphan drug development have over the last 12 years constituted a powerful impetus toward R&D directed at the rare diseases population.However, despite therapeutic promises contained in these projects and significant economic impact linked to burgeoning R&D expenditures, the affordability and value of OMPs has become a topic of health policy debate in Europe fueled by the perception that OMPs have high acquisition costs, and by misconceptions around pricing dynamics and rare-diseases business models. In order to maintain sustainable patient access to new and innovative therapies, it is essential to address these misconceptions, and to ensure the successful continuation of a dynamic OMPs R&D within rare-diseases public health policy.Misconceptions abound regarding the pricing of rare diseases drugs and reflect a poor appreciation of the R&D model and the affordability and value of OMPs.Simulation of potential financial returns of small medium sized rare diseases companies focusing on high priced drugs show that their economic returns are likely to be close to their cost of capital. Research in rare diseases is a challenging endeavour characterised by high fixed costs in which companies accrue substantial costs for several years before potentially generating returns from the fruits of their investments. Although heavily dependent upon R&D capabilities of each individual company or R&D organization, continuous flow of R&D financial investment should allow industry to increasingly include efficiencies in research and development in cost considerations to its customers. Industry should also pro-actively work on facilitating development of a specific value based pricing approach to help understanding what constitute value in rare diseases. Policy makers must reward innovation based upon unmet need and patient outcome. Broader understanding by clinicians, the public, and policy makers of the complexity of clinical programs to deliver OMPs to market is required to better comprehend the decisions needed and made by industry. In parallel, an overt effort to consider the impact of public policies on R&D investments is key to enable policy makers to better reconcile the incentives provided by public policy decisions and companies investments decisions in a more positive manner.

X Demographics

X Demographics

The data shown below were collected from the profiles of 7 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 150 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Brazil 2 1%
United Kingdom 1 <1%
Portugal 1 <1%
United States 1 <1%
Unknown 145 97%

Demographic breakdown

Readers by professional status Count As %
Student > Master 28 19%
Researcher 22 15%
Student > Ph. D. Student 16 11%
Other 10 7%
Student > Bachelor 9 6%
Other 32 21%
Unknown 33 22%
Readers by discipline Count As %
Medicine and Dentistry 34 23%
Pharmacology, Toxicology and Pharmaceutical Science 14 9%
Economics, Econometrics and Finance 10 7%
Social Sciences 9 6%
Agricultural and Biological Sciences 8 5%
Other 40 27%
Unknown 35 23%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 14. 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 27 December 2022.
All research outputs
#2,562,528
of 25,374,917 outputs
Outputs from Orphanet Journal of Rare Diseases
#325
of 3,105 outputs
Outputs of similar age
#21,320
of 209,223 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#3
of 46 outputs
Altmetric has tracked 25,374,917 research outputs across all sources so far. Compared to these this one has done well and is in the 89th percentile: it's in the top 25% 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 well, scoring higher than 89% 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 209,223 tracked outputs that were published within six weeks on either side of this one in any source. This one has done well, scoring higher than 89% of its contemporaries.
We're also able to compare this research output to 46 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 93% of its contemporaries.