Principle of protection and treatment of rare genetic diseases in Brazil: the case of lysosomal storage disorders

被引：6

作者：

Boy, Raquel ^{[1
]}

Schramm, Fermin Roland ^{[2
]}

机构：

[1] Univ Estado Rio de Janeiro, Dept Pediat, Ambulatorio Genet Clin, Fac Ciencias Med, BR-22250040 Rio De Janeiro, Brazil

[2] Fundacao Oswaldo Cruz, Escola Nacl Saude Publ Sergio Arouca, Rio De Janeiro, Brazil

来源：

CADERNOS DE SAUDE PUBLICA | 2009年 / 25卷 / 06期

关键词：

Lysossomal Storage Diseases; Exceptional Drugs; Bioethics; THERAPY; DRUGS;

D O I：

10.1590/S0102-311X2009000600010

中图分类号：

R1 [预防医学、卫生学];

学科分类号：

1004 ; 120402 ;

摘要：

This study aimed to discuss the morality of public funding for highly expensive orphan drugs for treatment of rare genetic diseases, using tools from bioethics, especially the principle of protection, applicable to vulnerable individuals and populations. Based on this principle, and considering the provisions of the Unified National Health System (SUS), the article argues for the state's moral obligation to provide public policies to ensure care for individuals with genetic diseases like lysosomal storage disorders, who can thus be viewed as "injured", besides suggesting measures to implement and ensure the sustainability of policies with an emphasis on resource allocation, targeting, and equity.

引用

页码：1276 / 1284

页数：9

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