Estimating the clinical cost of drug development for orphan versus non-orphan drugs
Estimating the clinical cost of drug development for orphan versus non-orphan drugs
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DOI：
10.1186/s13023-018-0990-4
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发表时间：
2019-01-10
影响因子：
3.7
通讯作者：
Grootendorst, Paul
中科院分区：
文献类型：
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作者：
Jayasundara, Kavisha;Hollis, Aidan;Grootendorst, Paul
背景孤儿药的高价已经引起了支付者和政策制定者的关注。这些价格可能反映了需要从一个小的病人库中收回药物开发的成本。然而，估计孤儿药开发的成本sparse.MethodsUsing公开数据，我们估计的差异，在试验的特点和临床开发成本与100孤儿和100非孤儿drugs.ResultsWe发现，自付临床成本每批准的孤儿药为1.66亿美元和2.91亿美元（2013年美元）每非孤儿药。每种获批孤儿药和非孤儿药的资本化临床成本估计分别为2.91亿美元和4.12亿美元。当专注于新的分子实体，我们发现，每批准的孤儿药的资本化临床成本是一半的非孤儿drug.ConclusionsMore讨论需要更好地调整成本组成部分应包括在研究和开发成本的药品。
BackgroundHigh orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the cost of orphan drug development are sparse.MethodsUsing publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs.ResultsWe found that the out-of-pocket clinical costs per approved orphan drug to be $166 million and $291 million (2013 USD) per non-orphan drug. The capitalized clinical costs per approved orphan drug and non-orphan drug were estimated to be $291 million and $412 million respectively. When focusing on new molecular entities only, we found that the capitalized clinical cost per approved orphan drug was half that of a non-orphan drug.ConclusionsMore discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.