Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products

British Journal of Clinical Pharmacology

Volumn 62, Issue 3, 2006, Pages 264-271

  Dear, James W ^a   Lilitkarntakul, Pajaree ^a   Webb, David J ^a  

^a UNIVERSITY OF EDINBURGH   (United Kingdom)

Author keywords

Orphan disease; Orphan medicinal product; Rare disease; Ultra orphan disease

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GLUCOSIDASE; ANAGRELIDE; ARSENIC TRIOXIDE; BOSENTAN; BUSULFAN; CARGLUMIC ACID; CELECOXIB; CLADRIBINE; IBUPROFEN; IDURONATE 2 SULFATASE; ILOPROST; IMATINIB; IMIGLUCERASE; LARONIDASE; LITAK; MIGLUSTAT; MITOTANE; NITISINONE; OGT 923; OMEGA CONOTOXIN MVIIA; ORPHAN DRUG; PEGVISOMANT; PHOTOFRIN; SILDENAFIL; UNCLASSIFIED DRUG; WILZIN; ZINC ACETATE;

ADENOMATOUS POLYP; ADRENAL CORTEX CARCINOMA; BARRETT ESOPHAGUS; BUDGET; CHRONIC MYELOID LEUKEMIA; CHRONIC PAIN; COST EFFECTIVENESS ANALYSIS; DRUG APPROVAL; DRUG COST; DRUG INDICATION; DRUG INDUSTRY; DRUG MARKETING; EUROPE; FABRY DISEASE; GASTROINTESTINAL STROMAL TUMOR; GAUCHER DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; HEALTH SERVICE; HUMAN; HUNTER SYNDROME; LAW; MUCOPOLYSACCHARIDOSIS; NIEMANN PICK DISEASE; NONHODGKIN LYMPHOMA; PATENT DUCTUS ARTERIOSUS; PRIORITY JOURNAL; PROMYELOCYTIC LEUKEMIA; PULMONARY HYPERTENSION; QUALITY ADJUSTED LIFE YEAR; RARE DISEASE; REVIEW; SANDHOFF DISEASE; THROMBOCYTHEMIA; THROMBOEMBOLISM; TYROSINEMIA; WILSON DISEASE;

DRUG DESIGN; DRUG INDUSTRY; HUMANS; MARKETING; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 33747050446     PISSN: 03065251     EISSN: 13652125     Source Type: Journal    
DOI: 10.1111/j.1365-2125.2006.02654.x     Document Type: Review

References (27)

1. Rinaldi A. Adopting an orphan. EMBO Report 2005; 6: 507-10.
2. Orphan Drug Act. http://www.fda.gov/orphan/oda.htm2005 Last accessed 13 December 2005.
3. European Committee for Orphan Medicinal Products. http://www.emea.eu. International/htms/general/contacts/COMP. html2005 Last accessed 13 December 2005.
4. Regulation (EC) no. 141/2000 of the European Parliament and the Council of 16 December 1999 on Orphan Medicinal Products. http://europa.eu.int/eur-lex/ lex/LexUriServ/LexUriServ.do?uri=CELEX.32000R0141:EN.HTML 2005 Last accessed 13 December 2005.
5. Loughnot D. Potential interactions of the Orphan Drug Act and pharmacogenomics: a flood of orphan drugs and abuses? Am J Law Med 2005; 31: 365-80.
6. Angus DC, Linde-Zwirble WT, Lidicker J, Clermont G, Carcillo J, Pinsky MR. Epidemiology of severe sepsis in the United States: analysis of incidence, outcome, and associated costs of care. Crit Care Med 2001; 29: 1303-10.
7. Arcaroli J, Fessler MB, Abraham E. Genetic polymorphisms and sepsis. Shock 2005; 24: 300-12.
8. Calvano SE, Xiao W, Richards DR, Felciano RM, Baker HV, Cho RJ, Chen RO, Brownstein BH, Cobb JP, Tschoeke SK, Miller-Graziano C, Moldawer LL, Mindrinos MN, Davis RW, Tompkins RG, Lowry SF. A network-based analysis of systemic inflammation in humans. Nature 2005; 437: 1032-7.
9. Fitzgerald JR, Sturdevant DE, Mackie SM, Gill SR, Musser JM. Evolutionary genomics of Staphylococcus aureus: insights into the origin of methicillin-resistant strains and the toxic shock syndrome epidemic. Proc Natl Acad Sci USA 2001; 98: 8821-6.
10. Haffner ME, Whitley J, Moses M. Two decades of orphan product development. Nat Rev Drug Discov 2002; 1: 821-5.
11. Reid B. New products highlight ambiguity of orphan drug law. Nat Biotechnol 2003; 21: 6-7.
12. Seget S. Orphans join European pharma family. Pharmafocus.com http://www.pharmafocus.com/cda/focusH/1,2109,22-0-0-0-focus_feature_detail-0- 353040,00.html 7-5-2005 Last accessed 13 December 2005.
13. Alcimed. Study on Orphan Drugs. Considerations on the Application of Article 8.2 of EC Regulation no. 141/2000 Concerning Orphan Drugs. http://pharmacos.eudra.org/F2/orphanmp/doc/pricestudy/ Final%20final%20report%20part%202%20web.pdf2005 Last accessed 13 December 2005.
14. Sheridan C. EU to review rare disease drugs market exclusivity. Nat Biotechnol 2004; 22: 1061.
15. Attina T, Camidge R, Newby DE, Webb DJ. Endothelin antagonism in pulmonary hypertension, heart failure, and beyond. Heart 2005; 91: 825-31.
16. Joppi R, Bertele V, Garrattini S. Orphan drug development is progressing too slowly. Br J Clin Pharmacol 2006; 61: 355-60.
17. Joppi R, Bertele V, Garattini S. Disappointing biotech. BMJ 2005; 331: 895-7.
18. Alcimed. Study on Orphan Drugs. Overview of the Conditions for Marketing Orphan Drugs in Europe. http://pharmacos.eudra.org/F2/orphanmp/doc/pricestudy/ Final%20final%20report%20part%201%20web.pdf2005 Last accessed 13 December 2005.
19. McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? BMJ 2005; 331: 1016-9.
20. European Platform for Patients' Organisations, Science and Industry EPPOSI. 4th EPPOSI Workshop on Partnering for Rare Disease Therapy Development. http://www.epposi.org/DenHague %20ProceedingsFinal%5B1%5D.pdf 2005 Last accessed 13 December 2005.
21. Phillip C, Thompson G. What is a QALY? http://www.evidencebased-medicine. co.uk/ebmfiles/WhatisaQALY.pdf2005 Last accessed 13 December 2005.
22. Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 2005; 98: 829-36.
23. Mckie J, Richardson J. The rule of rescue. Soc Sci Med 2003; 56: 2407-19.
24. NICE. Citizens Council Report Ultra Orphan Drugs. http://www.nice.org.uk/ pdf/boardmeeting/brdjan05item4.pdf2005 Last accessed 13 December 2005.
25. Department of Health. Cost Effective Provision of Disease-modifying Therapies for People with Multiple Sclerosis. http://www.dh.gov.uk/assetRoot/04/ 01/22/14/04012214.pdf4-2-2002 Last accessed 13 December 2005.
26. National Specialist Commissioning Advisory Group. http://www. advisorybodies.doh.gov.uk/NSCAG./index.htm2005 Last accessed 13 December 2005.
27. Burls A, Austin D, Moore D. Commissioning for rare diseases: view from the frontline. BMJ 2005; 331: 1019-21.