Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives

Intractable and Rare Diseases Research

Volumn 1, Issue 1, 2012, Pages 3-9

  Song, Peipei ^a   Gao, Jianjun ^a   Inagaki, Yoshinori ^a   Kokudo, Norihiro ^a   Tang, Wei ^a  

^a UNIVERSITY OF TOKYO   (Japan)

Author keywords

incentives; legislation; Orphan diseases; orphan drugs; reimbursement

Indexed keywords

EID: 84862339238     PISSN: 21863644     EISSN: 2186361x     Source Type: Journal    
DOI: 10.5582/irdr.2012.v1.1.3     Document Type: Article

References (33)

1. EURORDIS. What is a rare disease? http://www.eurordis.org (accessed December 8, 2011).
2. European Commission. Communication from the commission to the European parliament, the council, the European economic and social committee and the committee of the regions on rare diseases: Europe's challenges. http://ec.europa.eu/health/ph_threats/non_com/docs/rare_com_en.pdf (access December 8, 2011).
3. Stolk P, Willemen MJ, Leufkens H.G. Rare essentials: Drugs for rare diseases as essential medicines. Bull World Health Organ. 2006; 84:745-751.
4. EURORDIS. Rare diseases: Understanding this public health priority. http://www.eurordis.org/publication/rare-diseases-understanding-public-health-priority (access December 6, 2011).
5. Villa S, Compagni A, Reich M.R. Orphan drug legislation: Lessons for neglected tropical diseases. Int J Health Plann Manage. 2009; 24:27-42.
6. Taruscio D, Capozzoli F, Frank C. Rare diseases and orphan drugs. Ann Ist Super Sanita. 2011; 47:83-93.
7. Rinaldi A. Adopting an orphan. EMBO Rep. 2005; 6:507-510.
8. Ma D, Li DG, Zhang X, He L. The prevention and treatment on rare diseases in China: Opportunities and challenges. Chinese Journal of Evidence Based Pediatrics. 2011; 6:81-82. (in Chinese)
9. Lavandeira A. Orphan drugs: Legal aspects, current situation. Haemophilia. 2002; 8:194-198.
10. Committee for Orphan Medicinal Products and the European Medicines, Westermark K, Holm BB, et al. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nat Rev Drug Discov. 2011; 10:341-349.
11. Cheung RY, Cohen JC, Illingworth P. Orphan drug policies: Implications for the United States, Canada, and developing countries. Health Law J. 2004; 12:183-200.
12. Jaffe A, Zurynski Y, Beville L, Elliott E. Call for a national plan for rare diseases. J Paediatr Child Health. 2010; 46:2-4.
13. Schieppati A, Henter JI, Daina E, Aperia A. Why rare diseases are an important medical and social issue. Lancet. 2008; 371:2039-2041.
14. Park MH, Koo SK, Lee JS, Yoo HW, Kim JW, Cheong HI, Park H.Y. KMD: Korean mutation database for genes related to diseases. Hum Mutat. 2012. doi:10.1002/humu.22039. [Epub ahead of print]
15. Makoto S, Kiyohito N. Development of orphan drugs in Japan: Effects of a support system for development of orphan drugs in Japan. Drug Information Journal. 2000; 34:829-837.
16. Makoto S, Kiyohito N. Development of orphan drugs in Japan: Characteristics of orphan drugs developed in Japan. Drug Information Journal. 2000; 34:839-846.
17. Tambuyzer E. Rare diseases, orphan drugs and their regulation: Questions and misconceptions. Nat Rev Drug Discov. 2010; 9:921-929.
18. Schey C, Milanova T, Hutchings A. Estimating the budget impact of orphan medicines in Europe: 2010-2020. Orphanet J Rare Dis. 2011; 6:62.
19. Wellman-Labadie O, Zhou Y. The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity? Health Policy. 2010; 95:216-228.
20. Taiwan Foundation for rare disorders. About rare diseases. http://www.tfrd.org.tw (access December 9, 2011).
21. Kodama T, Tomita N. Global movement for diagnosis and treatment of rare/intractable diseases. J. Natl. Inst. Public Health. 2011; 60:105-111. (in Japanese)
22. Kanazawa I. Measures for dealing with “Intractable Diseases”-Past, present and future. ICORD 2012 Conference.
23. Ding JX, Sun XD, Ji N, Shao W.J. The accessibility assessment on orphan drugs and legal system research in China and America. Chinese Pharmaceutical Journal. 2011; 46:1129-1132. (in Chinese)
24. Lexchin J. Do manufacturers of brand-name drugs engage in price competition? An analysis of introductory prices. CMAJ. 2006; 174:1120-1121.
25. Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S. A comparative study of European rare disease and orphan drug markets. Health Policy. 2010; 97:173-179.
26. Japan Intractable Diseases Information Center. What is an intractable disease? http://www.nanbyou.or.jp (access December 9, 2011).
27. Liu BC, He L, He G, He Y. A cross-national comparative study of orphan drug policies in the United States, the European Union, and Japan: Towards a made-in-China orphan drug policy. J Public Health Policy. 2010; 31:407-420; discussion 420-421.
28. rare diseases in Shanghai could receive part of reimbursement. http://sh.eastday.com/qtmt/20111207/u1a944377.html (access December 9, 2011).
29. EURORDIS. Survey of the delay in diagnosis for 8 rare diseases in Europe (Eurordiscare 2). http://archive.eurordis.org/article.php3?id_article=454 (access February 1, 2012).
30. What is Rare Disease Day? http://www.rarediseaseday.org/article/why-rare-disease-day (access February 1, 2012).
31. Rare Diseases Clinical Research Network. Who are we? http://rarediseasesnetwork.epi.usf.edu (access February 1, 2012).
32. Aymé S, Kole A, Groft S. Empowerment of patients: Lessons from the rare diseases community. Lancet. 2008; 371:2048-2051.
33. Remuzzi G, Garattini S. Rare diseases: What's next? Lancet. 2008; 371:1978-1979.