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| Year : 2006 | Volume
: 38
| Issue : 5 | Page : 375 |
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Ultra orphan drugs and diseases
D Kumar, S Gupta
Department of Pharmacology and Therapeutics, Govt. Medical College Jammu. (J & K) - 180 001, India
Correspondence Address:
D Kumar
Department of Pharmacology and Therapeutics, Govt. Medical College Jammu. (J & K) - 180 001
India
 Source of Support: None, Conflict of Interest: None  | Check |
DOI: 10.4103/0253-7613.27716
How to cite this article:
Kumar D, Gupta S. Ultra orphan drugs and diseases. Indian J Pharmacol 2006;38:375 |
The educational forum, 'Orphan diseases and drugs', (Indian J Pharmacol 2006; 38:171-6) is a very informative article. The various aspects of orphan diseases and drugs have been well covered. A new and interesting term, 'ultra orphan drugs and diseases', was however not included in the article. Ultra orphan drugs[1] is the term designated to drugs which are used to treat exceptionally rare and chronically debilitating or life threatening diseases. Ultra orphan disease is the term designated to diseases with a prevalence of less than 0.18 case per 10,000 population.[2] A few examples of ultra orphan drugs/diseases are[3]
- Imantinib for gastrointestinal stromal tumours.
- Laronidase for mucopolysaccharidosis type 1.
- L-carnitine for genetic carnitine deficiency.
- Anagrelide for essential thrombocytopenia.
- Miglustat for Gaucher's disease.
- Imiglucerase for Gaucher's disease.
- Agalsidase for Fabry's disease.
- Orfadin for hereditary tyrosinaemia type 1.
- Sacrosidase for congenital sucrase isomaltase deficiency.
| References | |  |
| 1. | Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 2005;98:829-36.  [PUBMED] [FULLTEXT] |
| 2. | Rawlins M. Managing rare diseases. London: Royal College of Physicians; 2004. |
| 3. | McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? BMJ 2005;331:1016-9. [PUBMED] [FULLTEXT] |
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