遗传性血管性水肿：一种罕见病的治疗经济学
Hereditary Angioedema: The Economics of Treatment of an Orphan Disease.
作者信息
Lumry William Raymond
机构信息
Allergy Division, Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX, United States.
出版信息
Front Med (Lausanne). 2018 Feb 16;5:22. doi: 10.3389/fmed.2018.00022. eCollection 2018.
Abstract
This review will discuss the cost burden of hereditary angioedema on patients, healthcare systems, and society. The impact of availability of and access to novel and specific therapies on morbidity, mortality, and the overall burden of disease will be explored along with potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment. The prevalence of orphan diseases, legislative incentives to encourage development of orphan disease therapies and the impact of orphan disease treatment on healthcare payment systems will be discussed.
摘要
本综述将讨论遗传性血管性水肿给患者、医疗保健系统和社会带来的成本负担。还将探讨新型特异性疗法的可及性对发病率、死亡率和疾病总体负担的影响，以及治疗模式的潜在变化，以提高治疗效果并降低治疗成本。此外，还将讨论罕见病的患病率、鼓励罕见病治疗药物研发的立法激励措施以及罕见病治疗对医疗支付系统的影响。
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