Current Problems in Provision of Orphan Drugs and Ways to Solve Them
Despite the fact that orphan (rare) diseases are characterized by a very low prevalence rate, a considerable number of people worldwide suffer from these diseases. The United States and the European Union developed a variety of economic and administrative incentives for pharmaceutical companies to p...
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| Format: | Article |
| Language: | English |
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"Paediatrician" Publishers LLC
2016-09-01
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| Series: | Вопросы современной педиатрии |
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| Online Access: | https://vsp.spr-journal.ru/jour/article/view/1651 |
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| _version_ | 1850023672953176064 |
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| author | Alexei S. Kolbin Roman A. Gapeshin Stanislav M. Malyshev |
| author_facet | Alexei S. Kolbin Roman A. Gapeshin Stanislav M. Malyshev |
| author_sort | Alexei S. Kolbin |
| collection | DOAJ |
| description | Despite the fact that orphan (rare) diseases are characterized by a very low prevalence rate, a considerable number of people worldwide suffer from these diseases. The United States and the European Union developed a variety of economic and administrative incentives for pharmaceutical companies to produce orphan drugs, but their effectiveness is difficult to assess because of the small sample volume and complex organization of clinical trials. The average cost of orphan drugs is 5 times greater than that of the drugs used in the treatment of other diseases. Pricing mechanisms on orphan drugs are still poorly understood. |
| format | Article |
| id | doaj-art-e6797533f54b43649e697a6af757af0d |
| institution | DOAJ |
| issn | 1682-5527 1682-5535 |
| language | English |
| publishDate | 2016-09-01 |
| publisher | "Paediatrician" Publishers LLC |
| record_format | Article |
| series | Вопросы современной педиатрии |
| spelling | doaj-art-e6797533f54b43649e697a6af757af0d2025-08-20T03:01:18Zeng"Paediatrician" Publishers LLCВопросы современной педиатрии1682-55271682-55352016-09-0115434435110.15690/vsp.v15i4.15841612Current Problems in Provision of Orphan Drugs and Ways to Solve ThemAlexei S. Kolbin0Roman A. Gapeshin1Stanislav M. Malyshev2First Pavlov State Medical University of St. Petersburg, St. Petersburg, Russian Federation St. Petersburg State University, St. Petersburg, Russian FederationSt. Petersburg State University, St. Petersburg, Russian FederationFederal Almazov North-West Medical Research Centre, St. Petersburg, Russian FederationDespite the fact that orphan (rare) diseases are characterized by a very low prevalence rate, a considerable number of people worldwide suffer from these diseases. The United States and the European Union developed a variety of economic and administrative incentives for pharmaceutical companies to produce orphan drugs, but their effectiveness is difficult to assess because of the small sample volume and complex organization of clinical trials. The average cost of orphan drugs is 5 times greater than that of the drugs used in the treatment of other diseases. Pricing mechanisms on orphan drugs are still poorly understood.https://vsp.spr-journal.ru/jour/article/view/1651rare diseasesorphan drugsdevelopment |
| spellingShingle | Alexei S. Kolbin Roman A. Gapeshin Stanislav M. Malyshev Current Problems in Provision of Orphan Drugs and Ways to Solve Them Вопросы современной педиатрии rare diseases orphan drugs development |
| title | Current Problems in Provision of Orphan Drugs and Ways to Solve Them |
| title_full | Current Problems in Provision of Orphan Drugs and Ways to Solve Them |
| title_fullStr | Current Problems in Provision of Orphan Drugs and Ways to Solve Them |
| title_full_unstemmed | Current Problems in Provision of Orphan Drugs and Ways to Solve Them |
| title_short | Current Problems in Provision of Orphan Drugs and Ways to Solve Them |
| title_sort | current problems in provision of orphan drugs and ways to solve them |
| topic | rare diseases orphan drugs development |
| url | https://vsp.spr-journal.ru/jour/article/view/1651 |
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