Which ORPHANS Will Find a HOME? The Rule of Rescue in Resource Allocation for Rare Diseases
Which ORPHANS Will Find a HOME? The Rule of Rescue in Resource Allocation for Rare Diseases
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DOI：
10.1002/hast.12
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发表时间：
2012-01-01
影响因子：
3.3
通讯作者：
Pearson, Steven D.
中科院分区：
文献类型：
--
作者：
Largent, Emily A.;Pearson, Steven D.
救援规则描述了不惜一切代价拯救处于直接危险中的可识别生命的道德冲动。想想为营救掉进井里的小孩子、被压在地震废墟下的妇女或被困在海底的潜艇船员而采取的极端行动。再大的努力也不算太大。然而，这种不计成本进行救援的道德本能是否应该应用于急诊室、医院或社区诊所？在医疗保健领域，不惜一切代价拯救生命的愿望必须与资源稀缺的现实相协调。例如，预防因子VIII治疗一名血友病患者一年的估计费用为30万美元。发达国家的公共和私营保险公司已经接受了如此庞大的费用，尽管原则上，如果分配这些金额来支付许多其他患者未得到满足的医疗保健需求，这些金额可以提供更大的整体医疗福利。然而，展望未来，救援规则的广泛应用将越来越站不住脚。但道德本能仍将存在：帮助我们中最弱的人的愿望，特别是当他们的人数较少，让我们将他们视为独特的个体时。那么，什么是道德框架，可以指导未来的孤儿药物的覆盖和补偿决定？
The rule of rescue describes the moral impulse to save identifiable lives in immediate danger at any expense. Think of the extremes taken to rescue a small child who has fallen down a well, a woman pinned beneath the rubble of an earthquake, or a submarine crew trapped on the ocean floor. No effort is deemed too great. Yet should this same moral instinct to rescue, regardless of cost, be applied in the emergency room, the hospital, or the community clinic?In health care, the desire to save lives at any cost must be reconciled with the reality of resource scarcity. As one example, the estimated cost for prophylactic Factor VIII to treat one patient with hemophilia for one year is $300,000. Costs of this magnitude have been accepted by public and private insurers in the developed world, even though, in principle, these sums could provide greater overall health benefit if allocated to pay for the unmet health care needs of many other patients. Looking forward, however, broad application of the rule of rescue will be increasingly untenable. But the moral instinct will remain: the desire to help those weakest among us, especially when their small numbers allow us to see them as unique individuals. What, then, is the ethical framework that can guide coverage and reimbursement decisions for orphan drugs into the future?