Court-ordered access to treatment of rare genetic diseases: Fabry Disease in the state of Rio Grande do Sul, Brazil

Court-ordered access to high-cost drugs for rare genetic diseases, such as Fabry Disease (alpha-galactosidase-A deficiency), is a growing phenomenon as yet lacking systematic study. An observational, cross-sectional and retrospective study was conducted to characterize the lawsuits related to access...

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Bibliographic Details
Published in:Ciência & saude coletiva 2012-10, Vol.17 (10), p.2717-2728
Main Authors: Sartori Junior, Dailor, Leivas, Paulo Gilberto Cogo, Souza, Mônica Vinhas de, Krug, Bárbara Corrêa, Balbinotto, Giacomo, Schwartz, Ida Vanessa Doederlein
Format: Article
Language:Portuguese
Subjects:
alpha-Galactosidase - therapeutic use
beta-Galactosidase - therapeutic use
Brazil
Cross-Sectional Studies
Enzyme Replacement Therapy
Fabry Disease - drug therapy
Humans
Pharmaceutical Services - legislation & jurisprudence
Rare Diseases - drug therapy
Rare Diseases - genetics
Retrospective Studies
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Summary:Court-ordered access to high-cost drugs for rare genetic diseases, such as Fabry Disease (alpha-galactosidase-A deficiency), is a growing phenomenon as yet lacking systematic study. An observational, cross-sectional and retrospective study was conducted to characterize the lawsuits related to access to treatment for Fabry Disease by Enzyme Replacement Therapy in the State of Rio Grande do Sul prior to 2007. The study identified 13 lawsuits and 17 plaintiffs, 11 requesting alfa and 6 betagalsidase. The State of RS, the Federal Government, and 5 municipalities figured as defendants, in the form of joinder of parties or otherwise. There were 13 requests for interlocutory relief of which 12 were granted, and 2 sentences were handed down, both favorable. "Risk of death" was alleged by doctors in 4 prescriptions and by lawyers in the 13 lawsuits. The data suggest the lack of discussions combining aspects of medical efficacy and safety, cost-effectiveness, economic impact, and legal and constitutional arguments, which requires a specific policy for rare genetic diseases to standardize access to treatment.
ISSN:1678-4561
DOI:10.1590/S1413-81232012001000020