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

Journal of Medical Ethics

Volumn 37, Issue 4, 2011, Pages 233-239

  Boy, Raquel ^a   Schwartz, Ida V D ^b,c   Krug, Bárbara C ^c   Santana da Silva, Luiz C ^d   Steiner, Carlos E ^e   Acosta, Angelina X ^f   Ribeiro, Erlane M ^g   Galera, Marcial F ^h   Leivas, Paulo G C ⁱ   Braz, Marlene ^j  

^a STATE UNIVERSITY OF RIO DE JANEIRO   (Brazil)

^b HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^c FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

^d FEDERAL UNIVERSITY OF PARÁ   (Brazil)

^e UNIVERSITY OF CAMPINAS   (Brazil)

^f FEDERAL UNIVERSITY OF BAHIA   (Brazil)

^g Hospital Infantil Albert Sabin   (Brazil)

^h UNIVERSITY OF CUIABÁ   (Brazil)

ⁱ University Ritter dos Reis   (Brazil)

^j INSTITUTO OSWALDO CRUZ   (Brazil)

more..

Author keywords

[No Author keywords available]

Indexed keywords

LEVO IDURONIDASE;

ADOLESCENT; ADULT; AGE; AGED; ARTICLE; BRAZIL; CHILD; DRUG COST; DRUG MANUFACTURE; ECONOMICS; ETHICS; FEMALE; HEALTH CARE POLICY; HUMAN; HURLER SYNDROME; INFANT; LEGAL ASPECT; MALE; MIDDLE AGED; PRESCHOOL CHILD;

ADOLESCENT; ADULT; AGE FACTORS; AGED, 80 AND OVER; BRAZIL; CHILD; CHILD, PRESCHOOL; DRUG COSTS; FEMALE; HEALTH POLICY; HUMANS; IDURONIDASE; INFANT; MALE; MIDDLE AGED; MUCOPOLYSACCHARIDOSIS I; ORPHAN DRUG PRODUCTION; YOUNG ADULT;

EID: 79953316378     PISSN: 03066800     EISSN: 14734257     Source Type: Journal    
DOI: 10.1136/jme.2010.037150     Document Type: Article

References (36)

1. Neufeld E, Muenzer J. The mucopolysaccharidosis. In: Scriver CR, Beaudet AL, Sly S, et al, eds. The metabolic and molecular basis of inherited disease. 7th edn. New York, NY: McGraw-Hill, 2001:3421-52.
2. Meikle PJ, Hopwood JJ. Newborn screening for lysosomal storage: current issues and approaches. Curr Med Literature 2005;5:41-8.
3. Dear JW, Lilitkarntakul P, Webb DJ. Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products. Br J Pharmacol 2006;62:264-71.
4. Messina C, Rampazzo A, Cesaro S, et al. Eighteen-year follow-up of the first Italian MPS I patient treated with bone marrow transplantation. Bone Marrow Transplant 2008;41:905-6.
5. Anon. Drugs for rare diseases: mixed assessment in Europe. Prescrire Int 2007;16:36-42.
6. US Food and Drug Administration (FDA). Developing products for rare diseases and conditions. http://www.fda.gov/orphan/index.htm (accessed 15 Dec 2007).
7. European Medicines Agency (EMA). European Medicines Agency (EMA). Rare disease designation e Laronidase. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2009/11/human_orphan_000050.jsp&murl=menus/medicines/medicines.jsp&mid=WC0b01ac058001d12b (accessed 13 Jan 2011).
8. Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr 2004;144:581-8.
9. Schwartz I, Martins TF, Federhen A, et al. A Rede MPS faz quatro anos: um panorama geral sobre a epidemiologia das MPS no Brasil. In: Anais do XX Congresso Brasileiro de Genética Médica, 2008:21. Sociedade Brasileira de Genética Médica. Serrano Centro de Convenções, Gramado, RS, Brazil. 28 to 31 May 2008.
10. Agência Nacional de Vigilância Sanitária (ANVISA). Glossário da Anvisa. Brazil. http://e-glossario.bvs.br/glossary/public/scripts/php/page_search.php?lang=&letter=M (accessed 15 Dec 2007).
11. Goldim JR. O uso de drogas ainda experimentais em assistência: extensão de pesquisa, uso compassivo e acesso expandido. Rev Panam Salud Publica 2008;23:198-206.
12. Horovitz DD. Atenção aos defeitos congênitos no Brasil: proposta para estruturação e integração da abordagem no sistema de saúde [dissertation]. Rio de Janeiro, Brazil: Instituto de Medicina Social, Universidade do Estado do Rio de Janeiro. 2003.
13. Vieira T, Schwartz I, Muñoz V, et al. Mucopolysaccharidoses in Brazil: what happens from birth to biochemical diagnosis? Am J Med Genet A 2008;146A:1741-7.
14. Moore D, Connock MJ, Wraith E, et al. The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK. Orphanet J Rare Dis 2008;3:24.
15. Instituto Brasileiro de Geografia e Estatítica (IBGE). Contagem da população. Brazil, 2007. http://www.ibge.gov.br/home/estatistica/populacao/contagem2007/contagem.pdf (accessed Jan 2009).
16. Gericke CA, Riesberg A, Busse R. Ethical issues in funding orphan drug research and development. J Med Ethics 2005;31:164-8.
17. Beutler E. Lysosomal storage diseases: natural history and ethical and economic aspects. Mol Genet Metab 2006;88:208-15.
18. Connock M, Juarez-Garcia A, Few E, et al. A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies of Fabry's disease and mucopolysaccharidosis type I. Health Technol Assess 2006;10:iii-iv, ix-113.
19. Pastores GM, Arn P, Beck M, et al. The MPS I registry: design, methodology and early findings of a global disease registry for monitoring patients with mucopolysaccharidosis type I. Mol Genet Metab 2007;91:37-47.
20. Constituição da República Federativa do Brasil. Título VIII, capítulo II, seção II, "Da Saúde" (1988). http://www.planalto.gov.br/ccivil_03/constituicao/constitui%C3%A7ao.htm (accessed 12 Jan 2011).
21. Tocqueville A. A democracia na América. São Paulo: Abril, 1985:240.
22. Vieira FS, Zucchi P. Distorções causadas pelas ações judiciais à política de medicamentos no Brasil. Rev Saude Publica 2007;41:214-22.
23. Vieira FS. Ações judiciais e direito à saúde: reflexão sobre a observância aos princípios do SUS. Rev Saude Publica 2008;42:365-9.
24. Agência Nacional de Vigilância Sanitária (ANVISA), Brazil. Resolução n8 16, de 13 de março de 2008. Diário Oficial da União 2008;I:38.
25. Connock M, Burls A, Frew E, et al. The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review. Health Technol Assess 2006;10:iii-iv, ix-136.
26. Picon P, Prolla P, Amaral K, et al. Doença de Gaucher. In: Picon P, Beltrame A, eds. Protocolos clínicos e diretrizes terapêuticas. Brasília: Ministério da Sáude, 2002:217-34.
27. Wraith JE, Beck M, Lane R, et al. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years; results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Pediatrics 2007;120: e37-46.
28. Wraith JE, Hopwood JJ, Fuller M, et al. Laronidase treatment of mucopolysaccharidosis I. BioDrugs 2005;19:1-7.
29. Clarke LA, Wraith JE, Beck M, et al. Long term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics 2009;123:229-40.
30. Drummond MF, Wilson DA, Kanavos P, et al. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care 2007;23:36-42.
31. Raftery JP. Paying for costly pharmaceuticals: regulation of new drugs in Australia, England and New Zealand. Med J Aust 2008;188:26-8.
32. Beauchamp TL, Childress JF. Beneficence. In: Beauchamp TL, Childress JF, eds. Principles of biomedical ethics. Oxford: Oxford University Press, 2001:165-224.
33. Rawls J. Uma teoria da justiça. São Paulo: Martins Fontes, 1997.
34. Daniels N. Justice and justification: reflexive equilibrium in theory and practice. New York: Cambridge University Press, 1996.
35. Sen A. Desenvolvimento como Liberdade. São Paulo: Companhia das Letras, 2000:10.
36. Singer P. Vida Ética. Rio de Janeiro, Brazil: Ediouro, 2002.