Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I

Detalhes bibliográficos
Autor(a) principal: Boy, Raquel
Data de Publicação: 2011
Outros Autores: Schwartz, Ida Vanessa Doederlein, Krug, Bárbara C., Silva, Luiz C. Santana da, Steiner, Carlos Eduardo, Acosta, Angelina Xavier, Ribeiro, Erlane M., Galera, Marcial F., Leivas, Paulo G. C., Braz, Marlene
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UFBA
Texto Completo: http://repositorio.ufba.br/ri/handle/ri/17673
Resumo: Texto completo: acesso restrito. p. 233-239
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spelling Boy, RaquelSchwartz, Ida Vanessa DoederleinKrug, Bárbara C.Silva, Luiz C. Santana daSteiner, Carlos EduardoAcosta, Angelina XavierRibeiro, Erlane M.Galera, Marcial F.Leivas, Paulo G. C.Braz, MarleneBoy, RaquelSchwartz, Ida Vanessa DoederleinKrug, Bárbara C.Silva, Luiz C. Santana daSteiner, Carlos EduardoAcosta, Angelina XavierRibeiro, Erlane M.Galera, Marcial F.Leivas, Paulo G. C.Braz, Marlene2015-05-11T16:31:38Z20110306-6800http://repositorio.ufba.br/ri/handle/ri/17673v. 37, n. 4Texto completo: acesso restrito. p. 233-239Background/Aims Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disorder treated with bone marrow transplantation or enzyme replacement therapy with laronidase, a high-cost orphan drug. Laronidase was approved by the US Food and Drug Administration and the European Medicines Agency in 2003 and by the Brazilian National Health Surveillance Agency in 2005. Many Brazilian MPS I patients have been receiving laronidase despite the absence of a governmental policy regulating access to the drug. Epidemiological and treatment data concerning MPS I are scarce. This study aims to present a demographic profile of Brazilian patients with MPS I, describe the routes of access to laronidase in Brazil, and discuss associated ethical issues relating to public funding of orphan drugs. Methods In this cross-sectional observational study, data were collected nationwide between January and September 2008 from physicians, public institutions and non-governmental organisations involved with diagnosis and treatment of MPS I, using two data collection instruments specifically designed for this purpose. Results The minimum prevalence of MPS I in Brazil was estimated at 1/2 700 000. Most patients (69.8%) were younger than 15 years; 60 (88.2%) received laronidase. The most common route of access to the drug was through lawsuits (86.6%). Conclusions In Brazil, MPS I is predominantly a paediatric illness. Even though the cost of laronidase treatment is not officially covered by the Brazilian government, most MPS I patients receive the drug, usually through litigation. This gives rise to major ethical conflicts concerning drug access in a low-resource context. The Brazilian health policy framework lacks evidence-based clinical protocols for the distribution of orphan drugs.Submitted by Edileide Reis (leyde-landy@hotmail.com) on 2014-04-02T16:57:19Z No. of bitstreams: 1 Angelina X Acosta.pdf: 262279 bytes, checksum: 09fb34c293d14375f2eb9e9209994229 (MD5)Approved for entry into archive by Flávia Ferreira (flaviaccf@yahoo.com.br) on 2015-05-11T16:31:38Z (GMT) No. of bitstreams: 1 Angelina X Acosta.pdf: 262279 bytes, checksum: 09fb34c293d14375f2eb9e9209994229 (MD5)Made available in DSpace on 2015-05-11T16:31:38Z (GMT). No. of bitstreams: 1 Angelina X Acosta.pdf: 262279 bytes, checksum: 09fb34c293d14375f2eb9e9209994229 (MD5) Previous issue date: 2011http://dx.doi.org/ 10.1136/jme.2010.037150reponame:Repositório Institucional da UFBAinstname:Universidade Federal da Bahia (UFBA)instacron:UFBAMucopolysaccharidosisMucopolysaccharidosis ILaronidaseEthical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type IJournal of Medical Ethicsinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/article10000-01-01info:eu-repo/semantics/openAccessengLICENSElicense.txtlicense.txttext/plain1345https://repositorio.ufba.br/bitstream/ri/17673/2/license.txt0d4b811ef71182510d2015daa7c8a900MD52ORIGINALAngelina X Acosta.pdfAngelina X Acosta.pdfapplication/pdf262279https://repositorio.ufba.br/bitstream/ri/17673/1/Angelina%20X%20Acosta.pdf09fb34c293d14375f2eb9e9209994229MD51TEXTAngelina X Acosta.pdf.txtAngelina X Acosta.pdf.txtExtracted texttext/plain50284https://repositorio.ufba.br/bitstream/ri/17673/3/Angelina%20X%20Acosta.pdf.txt351168a2f254acb292a1034c4487dcfdMD53ri/176732022-08-26 11:02:39.622oai:repositorio.ufba.br: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Repositório InstitucionalPUBhttp://192.188.11.11:8080/oai/requestopendoar:19322022-08-26T14:02:39Repositório Institucional da UFBA - Universidade Federal da Bahia (UFBA)false
dc.title.pt_BR.fl_str_mv Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
dc.title.alternative.pt_BR.fl_str_mv Journal of Medical Ethics
title Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
spellingShingle Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
Boy, Raquel
Mucopolysaccharidosis
Mucopolysaccharidosis I
Laronidase
title_short Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
title_full Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
title_fullStr Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
title_full_unstemmed Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
title_sort Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
author Boy, Raquel
author_facet Boy, Raquel
Schwartz, Ida Vanessa Doederlein
Krug, Bárbara C.
Silva, Luiz C. Santana da
Steiner, Carlos Eduardo
Acosta, Angelina Xavier
Ribeiro, Erlane M.
Galera, Marcial F.
Leivas, Paulo G. C.
Braz, Marlene
author_role author
author2 Schwartz, Ida Vanessa Doederlein
Krug, Bárbara C.
Silva, Luiz C. Santana da
Steiner, Carlos Eduardo
Acosta, Angelina Xavier
Ribeiro, Erlane M.
Galera, Marcial F.
Leivas, Paulo G. C.
Braz, Marlene
author2_role author
author
author
author
author
author
author
author
author
dc.contributor.author.fl_str_mv Boy, Raquel
Schwartz, Ida Vanessa Doederlein
Krug, Bárbara C.
Silva, Luiz C. Santana da
Steiner, Carlos Eduardo
Acosta, Angelina Xavier
Ribeiro, Erlane M.
Galera, Marcial F.
Leivas, Paulo G. C.
Braz, Marlene
Boy, Raquel
Schwartz, Ida Vanessa Doederlein
Krug, Bárbara C.
Silva, Luiz C. Santana da
Steiner, Carlos Eduardo
Acosta, Angelina Xavier
Ribeiro, Erlane M.
Galera, Marcial F.
Leivas, Paulo G. C.
Braz, Marlene
dc.subject.por.fl_str_mv Mucopolysaccharidosis
Mucopolysaccharidosis I
Laronidase
topic Mucopolysaccharidosis
Mucopolysaccharidosis I
Laronidase
description Texto completo: acesso restrito. p. 233-239
publishDate 2011
dc.date.issued.fl_str_mv 2011
dc.date.accessioned.fl_str_mv 2015-05-11T16:31:38Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://repositorio.ufba.br/ri/handle/ri/17673
dc.identifier.issn.none.fl_str_mv 0306-6800
dc.identifier.number.pt_BR.fl_str_mv v. 37, n. 4
identifier_str_mv 0306-6800
v. 37, n. 4
url http://repositorio.ufba.br/ri/handle/ri/17673
dc.language.iso.fl_str_mv eng
language eng
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
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