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MeSH Review

Orphan Drug Production

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Disease relevance of Orphan Drug Production


High impact information on Orphan Drug Production

  • Preclinical and clinical evidence now indicate that meso-2,3-dimercaptosuccinic acid, an Orphan Drug, shows the most promise for being effective in this regard [6].
  • Dornase-alfa and orphan drugs [7].
  • Lithium: an orphan drug [8].
  • The federal Interagency Task Force on Drugs of Limited Commercial Value recommendations propose initiation of a wide spectrum of administrative and legislative changes; these are designed to create incentives for industry to cooperate with government in developing and making orphan drugs available to those who need them [9].
  • As a true "orphan drug" developed to treat a very small patient population, the cost per patient of PEG-ADA is very high [10].

Chemical compound and disease context of Orphan Drug Production


Associations of Orphan Drug Production with chemical compounds


Gene context of Orphan Drug Production

  • METHODS: Information about orphan drug designation and approval was obtained from the website of the European Commission-Enterprise and Industry DG and from the European Public Assessment Reports. RESULTS: Out of 255 OMP designations, only 18 were approved (7.1%) [16].
  • Orphan drugs and the NHS: should we value rarity [17]?
  • CMS move threatens to put orphan drugs out in cold [18].
  • The kallikrein inhibitor DX-88 (Dyax) has received orphan drug status in Europe and is undergoing clinical trial in Europe and the USA [19].


  1. A cost-effectiveness analysis of the orphan drug cysteamine in the treatment of infantile cystinosis. Soohoo, N., Schneider, J.A., Kaplan, R.M. Medical decision making : an international journal of the Society for Medical Decision Making. (1997) [Pubmed]
  2. Analysis of D-penicillamine by gas chromatography utilizing nitrogen--phosphorus detection. Rushing, L.G., Hansen, E.B., Thompson, H.C. J. Chromatogr. (1985) [Pubmed]
  3. 3,4-Diaminopyridine, an orphan drug, in the symptomatic treatment of Lambert-Eaton myasthenic syndrome. Molgó, J., Guglielmi, J.M. Pflugers Arch. (1996) [Pubmed]
  4. Development of an orphan drug by a start-up company. MetroGel for rosacea. Borgman, R.J. International journal of technology assessment in health care. (1992) [Pubmed]
  5. Gleevec for the treatment of chronic myelogenous leukemia: US. Food and Drug Administration regulatory mechanisms, accelerated approval, and orphan drug status. Cohen, M.H., Moses, M.L., Pazdur, R. Oncologist (2002) [Pubmed]
  6. meso-2,3-Dimercaptosuccinic acid: chemical, pharmacological and toxicological properties of an orally effective metal chelating agent. Aposhian, H.V., Aposhian, M.M. Annu. Rev. Pharmacol. Toxicol. (1990) [Pubmed]
  7. Dornase-alfa and orphan drugs. Collier, J. Lancet (1995) [Pubmed]
  8. Lithium: an orphan drug. Rosenthal, N.E. Arch. Gen. Psychiatry (2001) [Pubmed]
  9. Orphan drugs: creating a policy. Asbury, C.H., Stolley, P.D. Ann. Intern. Med. (1981) [Pubmed]
  10. PEG-ADA: an alternative to haploidentical bone marrow transplantation and an adjunct to gene therapy for adenosine deaminase deficiency. Hershfield, M.S. Hum. Mutat. (1995) [Pubmed]
  11. Topical metronidazole: a new therapy for rosacea. Schmadel, L.K., McEvoy, G.K. Clinical pharmacy. (1990) [Pubmed]
  12. Glycoprotein pharmaceuticals: scientific and regulatory considerations, and the US Orphan Drug Act. Liu, D.T. Trends Biotechnol. (1992) [Pubmed]
  13. Allergic reactions to Triatoma bites. Moffitt, J.E., Venarske, D., Goddard, J., Yates, A.B., deShazo, R.D. Ann. Allergy Asthma Immunol. (2003) [Pubmed]
  14. Methadone: an orphan drug? Ripamonti, C., Bianchi, M., Bruera, E. Journal of palliative medicine. (2004) [Pubmed]
  15. Imatinib. Novartis. Radford, I.R. Current opinion in investigational drugs (London, England : 2000) (2002) [Pubmed]
  16. Orphan drug development is progressing too slowly. Joppi, R., Bertele, V., Garattini, S. British journal of clinical pharmacology. (2006) [Pubmed]
  17. Orphan drugs and the NHS: should we value rarity? McCabe, C., Claxton, K., Tsuchiya, A. BMJ (2005) [Pubmed]
  18. CMS move threatens to put orphan drugs out in cold. Carroll, J. Managed care (Langhorne, Pa.) (2003) [Pubmed]
  19. Protease inhibitors in the treatment of hereditary angioedema. Ritchie, B.C. Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis. (2003) [Pubmed]
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