Jordan Bertrand
Biologiste, généticien et immunologiste, Président d'Aprogène (Association pour la promotion de la génomique), 13007 Marseille, France.
Med Sci (Paris). 2023 Feb;39(2):187-190. doi: 10.1051/medsci/2023003. Epub 2023 Feb 17.
After many years of development, a gene therapy for haemophilia B has demonstrated effectiveness and durability, at least over a time span of three years. It has just been approved (as Hemgenix®) by the FDA in the USA and by the EMA in the EU. However, the multimillion price tag of this treatment is a serious problem for healthcare systems and for patients, and raises a number of thorny issues about marketing practices of the pharmaceutical industry.
经过多年的发展，一种治疗乙型血友病的基因疗法已证明其有效性和持久性，至少在三年的时间跨度内如此。它刚刚获得美国食品药品监督管理局（FDA）和欧盟药品管理局（EMA）的批准（商品名为Hemgenix®）。然而，这种治疗方法高达数百万美元的价格标签对医疗保健系统和患者来说是一个严重问题，并引发了制药行业营销行为的诸多棘手问题。
