孤儿药的众筹：小皮娅的案例
Crowd Funding for Orphan Drugs: The Case of Baby Pia.
作者信息
Pinxten Wim
机构信息
Research Group Healthcare and Ethics, Faculty of Medicine and Life Sciences, Hasselt University, Hasselt, Belgium.
出版信息
Front Pharmacol. 2021 Dec 14;12:787342. doi: 10.3389/fphar.2021.787342. eCollection 2021.
Abstract
Medical crowdfunding is a relatively new strategy to obtain access to orphan drugs. The case of Baby Pia, a Belgian girl with SMA type 1 for whom in 2018 more than $ 2.1 million was raised to get her treated with Zolgensma, illustrates well the potential power of medical crowdfunding. But apart from the success in raising money, the case is also of particular importance for the ethical issues it brings to the surface as related to justice, equity, power imbalances, responsibility, accountability, indebtedness and privacy.
摘要
医疗众筹是获取罕见病药物的一种相对较新的策略。比利时女孩皮娅患有1型脊髓性肌萎缩症，2018年为了让她接受Zolgensma治疗，筹集了超过210万美元，皮娅的案例很好地说明了医疗众筹的潜在力量。但除了在筹集资金方面取得成功外，该案例还因其所引发的与正义、公平、权力失衡、责任、问责、债务和隐私相关的伦理问题而具有特别重要的意义。
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