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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]

Recent clinical studies

Etiology

Vassal G, Kearns P, Blanc P, Scobie N, Heenen D, Pearson A
Eur J Cancer 2017 Oct;84:149-158. Epub 2017 Sep 4 doi: 10.1016/j.ejca.2017.07.021. PMID: 28818704
Hughes DA, Poletti-Hughes J
PLoS One 2016;11(10):e0164681. Epub 2016 Oct 21 doi: 10.1371/journal.pone.0164681. PMID: 27768685Free PMC Article
Stockklausner C, Lampert A, Hoffmann GF, Ries M
Oncologist 2016 Apr;21(4):487-93. Epub 2016 Mar 28 doi: 10.1634/theoncologist.2015-0397. PMID: 27022038Free PMC Article
Hyry HI, Cox TM, Roos JC
Expert Rev Pharmacoecon Outcomes Res 2016;16(1):111-7. doi: 10.1586/14737167.2016.1141052. PMID: 26768506
Mechler K, Mountford WK, Hoffmann GF, Ries M
Orphanet J Rare Dis 2015 Apr 18;10:46. doi: 10.1186/s13023-015-0262-5. PMID: 25896727Free PMC Article

Diagnosis

Spagnolo P, du Bois RM, Cottin V
Lancet Respir Med 2013 Aug;1(6):479-87. Epub 2013 Jun 25 doi: 10.1016/S2213-2600(13)70085-7. PMID: 24429246
Hoffman EP, Connor EM
Discov Med 2013 Nov;16(89):233-9. PMID: 24229740
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. PMID: 21947805
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. PMID: 20566615
Arnon SS
Pediatrics 2007 Apr;119(4):785-9. doi: 10.1542/peds.2006-0646. PMID: 17403850

Therapy

Vassal G, Kearns P, Blanc P, Scobie N, Heenen D, Pearson A
Eur J Cancer 2017 Oct;84:149-158. Epub 2017 Sep 4 doi: 10.1016/j.ejca.2017.07.021. PMID: 28818704
Döring JH, Lampert A, Hoffmann GF, Ries M
PLoS One 2016;11(8):e0161660. Epub 2016 Aug 24 doi: 10.1371/journal.pone.0161660. PMID: 27557111Free PMC Article
Stockklausner C, Lampert A, Hoffmann GF, Ries M
Oncologist 2016 Apr;21(4):487-93. Epub 2016 Mar 28 doi: 10.1634/theoncologist.2015-0397. PMID: 27022038Free PMC Article
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. PMID: 23090701
Lesko LJ
Clin Pharmacol Ther 2012 Aug;92(2):258-61. Epub 2012 Jun 27 doi: 10.1038/clpt.2012.80. PMID: 22739138

Prognosis

Heslop E, Csimma C, Straub V, McCall J, Nagaraju K, Wagner KR, Caizergues D, Korinthenberg R, Flanigan KM, Kaufmann P, McNeil E, Mendell J, Hesterlee S, Wells DJ, Bushby K; TACT.
Orphanet J Rare Dis 2015 Apr 23;10:49. doi: 10.1186/s13023-015-0258-1. PMID: 25902795Free PMC Article
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. PMID: 21953319
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. PMID: 18210097Free PMC Article

Clinical prediction guides

Hughes DA, Poletti-Hughes J
PLoS One 2016;11(10):e0164681. Epub 2016 Oct 21 doi: 10.1371/journal.pone.0164681. PMID: 27768685Free PMC Article
Döring JH, Lampert A, Hoffmann GF, Ries M
PLoS One 2016;11(8):e0161660. Epub 2016 Aug 24 doi: 10.1371/journal.pone.0161660. PMID: 27557111Free PMC Article
Stockklausner C, Lampert A, Hoffmann GF, Ries M
Oncologist 2016 Apr;21(4):487-93. Epub 2016 Mar 28 doi: 10.1634/theoncologist.2015-0397. PMID: 27022038Free PMC Article
Pastores GM, Gupta P
Pediatr Endocrinol Rev 2013 Nov;11 Suppl 1:64-7. PMID: 24380124
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. PMID: 18210097Free PMC Article

Recent systematic reviews

Stockklausner C, Lampert A, Hoffmann GF, Ries M
Oncologist 2016 Apr;21(4):487-93. Epub 2016 Mar 28 doi: 10.1634/theoncologist.2015-0397. PMID: 27022038Free PMC Article
Gammie T, Lu CY, Babar ZU
PLoS One 2015;10(10):e0140002. Epub 2015 Oct 9 doi: 10.1371/journal.pone.0140002. PMID: 26451948Free PMC Article
Richter T, Nestler-Parr S, Babela R, Khan ZM, Tesoro T, Molsen E, Hughes DA; International Society for Pharmacoeconomics and Outcomes Research Rare Disease Special Interest Group.
Value Health 2015 Sep;18(6):906-14. Epub 2015 Aug 18 doi: 10.1016/j.jval.2015.05.008. PMID: 26409619
Heslop E, Csimma C, Straub V, McCall J, Nagaraju K, Wagner KR, Caizergues D, Korinthenberg R, Flanigan KM, Kaufmann P, McNeil E, Mendell J, Hesterlee S, Wells DJ, Bushby K; TACT.
Orphanet J Rare Dis 2015 Apr 23;10:49. doi: 10.1186/s13023-015-0258-1. PMID: 25902795Free PMC Article
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. PMID: 23090701

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