| Title |
Issues surrounding orphan disease and orphan drug policies in Europe
|
|---|---|
| Published in |
Applied Health Economics and Health Policy, August 2012
|
| DOI | 10.2165/11536990-000000000-00000 |
| Pubmed ID | |
| Authors |
Alain Denis, Lut Mergaert, Christel Fostier, Irina Cleemput, Steven Simoens |
| Abstract |
An orphan disease is a disease with a very low prevalence. Although there are 5000-7000 orphan diseases, only 50 orphan drugs (i.e. drugs developed to treat orphan diseases) were marketed in the EU by the end of 2008. In 2000, the EU implemented policies specifically designed to stimulate the development of orphan drugs. While decisions on orphan designation and the marketing authorization of orphan drugs are made at the EU level, decisions on drug reimbursement are made at the member state level. The specific features of orphan diseases and orphan drugs make them a high-priority issue for policy makers. The aim of this article is to identify and discuss several issues surrounding orphan disease and drug policies in Europe. The present system of orphan designation allows for drugs for non-orphan diseases to be designated as orphan drugs. The economic factors underlying orphan designation can be questioned in some cases, as a low prevalence of a certain indication does not equal a low return on investment for the drug across its indications. High-quality evidence about the clinical added value of orphan drugs is rarely available at the time of marketing authorization, due to the low number of patients. A balance must be struck between ethical and economic concerns. To this effect, there is a need to initiate a societal dialogue on this issue, to clarify what society wants and accepts in terms of ethical and economic consequences. The growing budgetary impact of orphan drugs puts pressure on drug expenditure. Indications can be extended for an orphan drug and the total prevalence across indications is not considered. Finally, cooperation needs to be fostered in the EU, particularly through a standardized approach to the creation and use of registries. These issues require further attention from researchers, policy makers, health professionals, patients, pharmaceutical companies and other stakeholders with a view to optimizing orphan disease and drug policies in Europe. |
Mendeley readers
The data shown below were compiled from readership statistics for 107 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 2 | 2% |
| Brazil | 2 | 2% |
| Canada | 1 | <1% |
| Spain | 1 | <1% |
| Unknown | 101 | 94% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Master | 31 | 29% |
| Student > Bachelor | 16 | 15% |
| Researcher | 13 | 12% |
| Student > Doctoral Student | 8 | 7% |
| Student > Ph. D. Student | 7 | 7% |
| Other | 20 | 19% |
| Unknown | 12 | 11% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 34 | 32% |
| Social Sciences | 13 | 12% |
| Pharmacology, Toxicology and Pharmaceutical Science | 8 | 7% |
| Agricultural and Biological Sciences | 7 | 7% |
| Nursing and Health Professions | 6 | 6% |
| Other | 28 | 26% |
| Unknown | 11 | 10% |
Attention Score in Context
This research output has an Altmetric Attention Score of 6. 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 25 November 2021.
All research outputs
#5,446,629
of 25,373,627 outputs
Outputs from Applied Health Economics and Health Policy
#247
of 841 outputs
Outputs of similar age
#37,592
of 182,990 outputs
Outputs of similar age from Applied Health Economics and Health Policy
#13
of 82 outputs
Altmetric has tracked 25,373,627 research outputs across all sources so far. Compared to these this one has done well and is in the 75th percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 841 research outputs from this source. They typically receive more attention than average, with a mean Attention Score of 9.3. This one has gotten more attention than average, scoring higher than 68% of its peers.
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We're also able to compare this research output to 82 others from the same source and published within six weeks on either side of this one. This one has gotten more attention than average, scoring higher than 68% of its contemporaries.