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新型治疗产品与艰难抉择。以重组凝血因子VIII治疗甲型血友病为例

New therapeutic products and difficult decisions. The case of recombinant factor VIII in the management of haemophilia A.

作者信息

Green C, Akehurst R

机构信息

School of Health and Related Research, University of Sheffield.

出版信息

J Public Health Med. 1998 Jun;20(2):137-8. doi: 10.1093/oxfordjournals.pubmed.a024732.

DOI:10.1093/oxfordjournals.pubmed.a024732
PMID:9675729
Abstract

New and expensive therapeutic products can place great pressure on health care purchasers. Often, evidence to support the inclusion of such products in the purchasing process is lacking or confusing, yet demand can be organized and forceful. In this paper we use the example of the introduction of recombinant factor VIII (rFVIII) in the management of haemophilia A, to highlight some problems purchasers face in deciding whether to fund its use. The introduction of rFVIII involves substantial extra funding requirements and the benefits afforded by its use are unclear. Although a case can be made for the use of rFVIII on the grounds of subjective furture theoretical risks and intangible benefits, the purchasers of health care are charged with maximizing the present and obtainable benefits of the community at large, given finite resources. Guidelines produced by the United Kingdom Haemophilia Centre Directors Organization state rFVIII to be its treatment of choice for all haemophilia A patients, but offer no insight into the benefits attainable through the use of rFVIII, in terms of health outcomes. We report summary findings of Purchaser Intelligence Groups examining the use of rFVIII and offer comment on the problems associated with the implementation of therapeutic products such as rFVIII.

摘要

新型且昂贵的治疗产品会给医疗保健采购方带来巨大压力。通常,在采购过程中支持纳入此类产品的证据要么缺乏,要么令人困惑,但需求可能既成规模又很迫切。在本文中,我们以重组因子VIII(rFVIII)用于治疗A型血友病为例,来突出采购方在决定是否资助其使用时所面临的一些问题。引入rFVIII需要大量额外资金,且其使用所带来的益处尚不明确。尽管基于主观的未来理论风险和无形益处,可以为使用rFVIII提出理由,但在资源有限的情况下,医疗保健采购方的职责是使整个社会的当前和可获得利益最大化。英国血友病中心主任组织制定的指南指出,rFVIII是所有A型血友病患者的首选治疗方法,但就健康结果而言,并未深入探讨使用rFVIII可获得的益处。我们报告了采购方情报小组对rFVIII使用情况的总结调查结果,并对与rFVIII等治疗产品实施相关的问题发表评论。

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