Does orphan drug legislation really answer the needs of patients?

Abstract

References 1 van Weely S, Leufkens HGM. Orphan diseases. Background paper. In: priority medicines for Europe and the world. A public health approach to innovation. http://mednet3.who.int/ prioritymeds/report/index.htm#c (accessed July, 2006). 2 US Food and Drug Administration. Orphan Drug Act, Pub L. No. 97-414 , 96 Stat. 2049. 1982. http://www.fda.gov/orphan/oda.htm (accessed May, 2008). 3 European Commission. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. http://ec.europa.eu/enterprise/ pharmaceuticals/eudralex/homev1.htm (accessed July, 2006). 4 OrphanXchange. About orphan drugs. http://www. orphanxchange.org/OXC/cgi-bin/oxc_about_drug.php (accessed July, 2006). 5 Wastfelt M, Fadeel B, Henter J-I. A journey of hope: lessons learned from studies on rare diseases and orphan drugs. J Intern Med 2006; 260: 1–10. 6 United States Department of Health and Human Services, Public Health Service, Offi ce of the Assistant Secretary for Health. Report of the National Commission on Orphan Diseases. Rockville, MD: Offi ce of the Assistant Secretary for Health, 1989. 7 WHO. International Classifi cation of Diseases. 10th revision. http://www.who.int/classifi cations/apps/icd/icd10online/ (accessed July, 2006). 8 Orphanet. Rare diseases in numbers: preliminary report from an on going bibliographic study initiated by Eurordis in partnership with Orphanet. http://www.orpha.net/actor/Orphanews/2005/ doc/Rare_Diseases_in_Numbers.pdf (accessed July, 2006). 9 DL Weiner. Pediatrics: inborn errors of metabolism. http://www. emedicine.com/emerg/topic768.htm (accessed July, 2006). 10 Dionisi-Vici C, Rizzo C, Burlina AB, et al. Inborn errors of metabolism in the Italian pediatric population: a national retrospective survey. J Pediatr 2002; 140: 321–27. 11 Eurordis. EurordisCare2: survey of diagnostic delays, 8 diseases, Europe. http://www.eurordis.org/big_article.php3?id_article=454 (accessed July, 2006). 12 The Independent Expert Group. Ethical, legal and social aspects of genetic testing: research, development and clinical applications. Brussels, 2004. http://ec.europa.eu.research/ conferences/2004/genetic/report_en.htm (accessed July, 2006). 13 Overview of current Centres of Reference on rare diseases in the EU. Report to the High Level Group on Health Services and Medical Care, and Annexes. September 2005. http://ec.europa. eu/health/ph_information/implement/wp/morbidity/keydocs_ morbidity_en.htm (accessed July, 2006). 14 Haff ner ME. Adopting orphan drugs: two dozen years of treating rare diseases. N Engl J Med 2006; 354: 445–47.

DOI: 10.1016/S0140-6736(08)60873-9
010002000300020102011201220132014201520162017
Citations per Year

5,154 Citations

Semantic Scholar estimates that this publication has 5,154 citations based on the available data.

See our FAQ for additional information.

Cite this paper

@article{Haffner2008DoesOD, title={Does orphan drug legislation really answer the needs of patients?}, author={Marlene E. Haffner and Josep Torrent-Farnell and Paul D Maher}, journal={Lancet}, year={2008}, volume={371 9629}, pages={2041-4} }