| Literature DB >> 35366215 |
Hong Si Nga1, Lilian Monteiro Pereira Palma2, Miguel Ernandes Neto3, Luis Gustavo Modelli de Andrade4.
Abstract
Atypical hemolytic uremic syndrome (aHUS) is an ultra-rare disease and eculizumab was approved as first line therapy in 2011 by the Food and Drug Administration. Access to eculizumab in low-middle income countries is challenging. We discuss access to eculizumab in Brazil that was made possible by judicialization or compassionate use. We showed a nationwide cohort of unplanned eculizumab interruptions resulted in higher rates of aHUS relapse. Similar to the French cohort, the use of eculizumab after transplantation showed superior graft survival compared to conventional treatment. We speculate a possible solution to the higher cost of eculizumab in which the government negotiates with the manufacturers. In this process, the government should compromise to ensure validated protocols of drug use, and the pharmaceutical companies, on the other hand, should reduce prices, especially in low-income countries. We also suggest a price adjustment based on gross domestic product.Entities:
Keywords: Atypical hemolytic uremic syndrome; Eculizumab; Low-middle income countries
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Year: 2022 PMID: 35366215 DOI: 10.1007/s40620-022-01282-4
Source DB: PubMed Journal: J Nephrol ISSN: 1121-8428 Impact factor: 3.902