[Principle of protection and treatment of rare genetic diseases in Brazil: the case of lysosomal storage disorders].
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.
Boy
Raquel
R
Departamento de Pediatria, Ambulatório de Genética Clínica, Faculdade de Ciências Médicas, Universidade do Estado do Rio de Janeiro, Rio de Janeiro, Brasil. raquelboy@ig.com.br
Schramm
Fermin Roland
FR
D001675:Bioethics; D001938:Brazil; D016265:Conflict of Interest; D004358:Drug Therapy / Q000191:economics / Q000941:ethics; D035082:Federal Government; D006801:Humans; D007880:Legislation, Drug; D016464:Lysosomal Storage Diseases / Q000188:drug therapy*; D009014:Morals; D009313:National Health Programs / Q000941:ethics*; D009965:Orphan Drug Production / Q000191:economics / Q000941:ethics* / Q000331:legislation & jurisprudence; D028662:Principle-Based Ethics; D011640:Public Policy; D035583:Rare Diseases / Q000188:drug therapy*; D012935:Social Justice; D035862:Vulnerable Populations