2010
DOI: 10.1590/s1413-81232010000900019
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Abstract: Este artigo aborda, de forma crítica, aspectos das políticas públicas brasileiras para medicamentos, com ênfase nos de alto custo dirigidos às doenças raras. As doenças lisossômicas foram utilizadas como exemplo pela sua raridade e pela tendência mundial para o desenvolvimento de novos fármacos para seu tratamento. Três doenças foram abordadas: doença de Gaucher, doença de Fabry e mucopolissacaridose tipo I. Embora todas tenham medicamentos registrados no Brasil, a doença de Gaucher é a única com protocolo clí… Show more

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Cited by 28 publications
(22 citation statements)
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“…Indeed, in 2007, about 500 patients with GD type 1 were treated in Brazil with 177,280 bottles of ERT being purchased at a cost of over US$ 125 million (Souza et al 2010). The effective dose of imiglucerase is individually adjusted according to the clinical status and treatment response of the patient.…”
Section: Introductionmentioning
confidence: 99%
“…Indeed, in 2007, about 500 patients with GD type 1 were treated in Brazil with 177,280 bottles of ERT being purchased at a cost of over US$ 125 million (Souza et al 2010). The effective dose of imiglucerase is individually adjusted according to the clinical status and treatment response of the patient.…”
Section: Introductionmentioning
confidence: 99%
“…Contudo, tais incentivos estimulam o monopólio de indústrias farmacêuticas, o que torna os medicamentos mais lucrativos para elas, deixando as pessoas com doenças raras vulneráveis e sem perspectivas de tratamento (SOUZA et al 2010 Nesse contexto, as pessoas com doenças raras tornam-se uma mina de ouro para a indústria farmacêutica caso a mesma consiga os benefícios do governo, como os subsídios federais, os incentivos fiscais e a exclusividade de marketing, que no caso das doenças raras é de sete anos para o tratamento da doença (DALLARI, 2015). Assim, o desenvolvimento de fármacos, mesmo que para um pequeno número de clientes, pode gerar lucros em milhões de dólares.…”
Section: Os Medicamentos Para Tratamento Das Doenças Raras E O Papel unclassified
“…According to the Brazilian Health Surveillance Agency (ANVISA) 15 , "Rare or orphan diseases are those that affect small numbers of persons within the general population." this definition exists in order to supply the specific drugs 16,17 . However, the Brazilian Ministry of Health has already developed and published Clinical protocols and therapeutic Guidelines for 26 rare diseases, including amyotrophic lateral sclerosis, congenital adrenal hyperplasia, Guillain-Barré syndrome, Gaucher's disease, and Wilson's disease, among others.…”
Section: Country/regionmentioning
confidence: 99%
“…An example is type I mucopolysaccharidosis, a lysosomal storage disease caused by deficient activity in the enzyme αL-iduronidase (and with an estimated 87 patients identified in Brazil) 16 . If the principle of supplying the drug to research subjects were enforced, and if this clinical trial had a representative sample size, practically all the potential demand for the drug would be consumed by post-trial access for the study volunteers.…”
Section: Rare Diseases and Research Ethicsmentioning
confidence: 99%
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