Resumo

HEYERDAHL-VIAU, Ivo  e  LOPEZ-NARANJO, Francisco. Hemgenix®, the most expensive drug in the world in 2023. Rev Hum Med [online]. 2024, vol.24, n.2  Epub 29-Jun-2024. ISSN 1727-8120.

The aim of this bibliographic review is to describe the function of Hemgenix®, the most expensive drug in the world in 2023, with the purpose of providing current information to the line of research of Clinical Pharmacy and Pharmaceutical Services, especially in the area of orphan drugs. For this purpose, a bibliographic search was carried out in October 2023, in the Department of Biological Systems, Metropolitan Autonomous University Xochimilco Unit, in Mexico City. Hemgenix® is a gene therapy used to cure hemophilia B, which is a rare disease and whose current treatment, although effective, does not completely cures the disease and must be administered for life, which generates a great economic burden. Hemgenix®, approved in the United States in November 2023, is presented with the support of a clinical trial that indicates that a dose costing US$3.5 M can practically eradicate the disease. However, there is still much uncertainty regarding its long-term safety and effectiveness, making its cost-effectiveness assessments uncertain for health systems that must make decisions to manage and make the best use of their limited health resources.

Palavras-chave : hemophilia B; rare diseases; genetic therapy; health expenditure.

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