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Orphan drug

MedGen UID:
4410
Concept ID:
C0013232
Pharmacologic Substance
Synonyms: Drug, Orphan; Drugs, Orphan; Orphan Drug; Orphan Drugs
SNOMED CT: Orphan drug (40606008)
 

Definition

A compound or product that demonstrates promise for the diagnosis and/or treatment of rare diseases and conditions that affect fewer than 200,000 people. The U.S. Orphan Drug Act (1983) encourages development of these therapeutics by tax breaks and a seven-year monopoly on drug sales. These incentives promote the development and manufacturing of such therapeutics, which otherwise might not be profitable because of the small potential market. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews
  • CROGOrphan drug

Recent clinical studies

Etiology

Manuel J, Collin-Histed T
Pediatr Endocrinol Rev 2013 Nov;11 Suppl 1:116-24. PMID: 24380130
Hoffman EP, Connor EM
Discov Med 2013 Nov;16(89):233-9. PMID: 24229740
Hughes-Wilson W, Palma A, Schuurman A, Simoens S
Orphanet J Rare Dis 2012 Sep 26;7:74. doi: 10.1186/1750-1172-7-74. [Epub ahead of print] PMID: 23013790Free PMC Article
Kesselheim AS
Clin Pharmacol Ther 2012 Aug;92(2):153-5. doi: 10.1038/clpt.2012.92. PMID: 22814660
von Stülpnagel C, Coppola G, Striano P, Müller A, Staudt M, Kluger G
Eur J Paediatr Neurol 2012 Sep;16(5):459-63. Epub 2012 Jan 21 doi: 10.1016/j.ejpn.2011.12.012. [Epub ahead of print] PMID: 22266062

Diagnosis

Hoffman EP, Connor EM
Discov Med 2013 Nov;16(89):233-9. PMID: 24229740
Mueller SM, Itin P, Haeusermann P
Skinmed 2013 Mar-Apr;11(2):80-3. PMID: 23745225
Pinxten W, Denier Y, Dooms M, Cassiman JJ, Dierickx K
J Med Ethics 2012 Mar;38(3):148-53. Epub 2011 Sep 24 doi: 10.1136/medethics-2011-100094. [Epub ahead of print] PMID: 21947805
Goldsborough AS, Handley MD, Dulcey AE, Pluchino KM, Kannan P, Brimacombe KR, Hall MD, Griffiths G, Gottesman MM
J Med Chem 2011 Jul 28;54(14):4987-97. Epub 2011 Jun 24 doi: 10.1021/jm2001663. [Epub ahead of print] PMID: 21657271Free PMC Article
Talele SS, Xu K, Pariser AR, Braun MM, Farag-El-Massah S, Phillips MI, Thompson BH, Coté TR
Pediatrics 2010 Jul;126(1):101-6. Epub 2010 Jun 21 doi: 10.1542/peds.2009-3246. [Epub ahead of print] PMID: 20566615

Therapy

Hoffman EP, Connor EM
Discov Med 2013 Nov;16(89):233-9. PMID: 24229740
Mueller SM, Itin P, Haeusermann P
Skinmed 2013 Mar-Apr;11(2):80-3. PMID: 23745225
Joppi R, Bertele' V, Garattini S
Eur J Clin Pharmacol 2013 Apr;69(4):1009-24. Epub 2012 Oct 23 doi: 10.1007/s00228-012-1423-2. [Epub ahead of print] PMID: 23090701
Kesselheim AS
Clin Pharmacol Ther 2012 Aug;92(2):153-5. doi: 10.1038/clpt.2012.92. PMID: 22814660
Lesko LJ
Clin Pharmacol Ther 2012 Aug;92(2):258-61. Epub 2012 Jun 27 doi: 10.1038/clpt.2012.80. [Epub ahead of print] PMID: 22739138

Prognosis

Pastores GM, Gupta P
Pediatr Endocrinol Rev 2013 Nov;11 Suppl 1:64-7. PMID: 24380124
Lichtenberg FR
Eur J Health Econ 2013 Feb;14(1):41-56. Epub 2011 Sep 28 doi: 10.1007/s10198-011-0349-4. [Epub ahead of print] PMID: 21953319
Schey C, Milanova T, Hutchings A
Orphanet J Rare Dis 2011 Sep 27;6:62. doi: 10.1186/1750-1172-6-62. [Epub ahead of print] PMID: 21951518Free PMC Article
Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S
Appl Health Econ Health Policy 2010;8(5):343-50. doi: 10.2165/11536990-000000000-00000. PMID: 20804226
Heemstra HE, de Vrueh RL, van Weely S, Büller HA, Leufkens HG
Eur J Clin Pharmacol 2008 May;64(5):545-52. Epub 2008 Jan 22 doi: 10.1007/s00228-007-0454-6. [Epub ahead of print] PMID: 18210097Free PMC Article

Clinical prediction guides

Pastores GM, Gupta P
Pediatr Endocrinol Rev 2013 Nov;11 Suppl 1:64-7. PMID: 24380124
von Stülpnagel C, Coppola G, Striano P, Müller A, Staudt M, Kluger G
Eur J Paediatr Neurol 2012 Sep;16(5):459-63. Epub 2012 Jan 21 doi: 10.1016/j.ejpn.2011.12.012. [Epub ahead of print] PMID: 22266062
Schey C, Milanova T, Hutchings A
Orphanet J Rare Dis 2011 Sep 27;6:62. doi: 10.1186/1750-1172-6-62. [Epub ahead of print] PMID: 21951518Free PMC Article
Kluger G, Haberlandt E, Kurlemann G, Ernst JP, Runge U, Schneider F, Makowski C, Boor R, Bast T
Epilepsy Behav 2010 Apr;17(4):546-8. Epub 2010 Feb 24 doi: 10.1016/j.yebeh.2010.01.005. [Epub ahead of print] PMID: 20185372
Heemstra HE, de Vrueh RL, van Weely S, Büller HA, Leufkens HG
Eur J Clin Pharmacol 2008 May;64(5):545-52. Epub 2008 Jan 22 doi: 10.1007/s00228-007-0454-6. [Epub ahead of print] PMID: 18210097Free PMC Article

Recent systematic reviews

Kanters TA, de Sonneville-Koedoot C, Redekop WK, Hakkaart L
Orphanet J Rare Dis 2013 Aug 16;8:124. doi: 10.1186/1750-1172-8-124. [Epub ahead of print] PMID: 23947946Free PMC Article
Mansour H, Chahine EB, Karaoui LR, El-Lababidi RM
Ann Pharmacother 2013 Mar;47(3):368-79. Epub 2013 Mar 5 doi: 10.1345/aph.1R435. [Epub ahead of print] PMID: 23463743
Joppi R, Bertele' V, Garattini S
Eur J Clin Pharmacol 2013 Apr;69(4):1009-24. Epub 2012 Oct 23 doi: 10.1007/s00228-012-1423-2. [Epub ahead of print] PMID: 23090701
Kesselheim AS, Myers JA, Avorn J
JAMA 2011 Jun 8;305(22):2320-6. doi: 10.1001/jama.2011.769. PMID: 21642684
McCabe C, Edlin R, Round J
Adv Exp Med Biol 2010;686:211-22. doi: 10.1007/978-90-481-9485-8_13. PMID: 20824448

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