Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Canada.
Department of Economics, University of Calgary, Calgary, Canada.
Orphanet J Rare Dis. 2019 Jan 10;14(1):12. doi: 10.1186/s13023-018-0990-4.
High 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.
Using publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs.
We 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.
More discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.
高孤儿药价格引起了支付者和政策制定者的关注。这些价格可能反映了从一个小患者群体中收回药物开发成本的需要。然而，孤儿药开发成本的估计数据很少。
我们使用公开数据，估计了 100 种孤儿药和 100 种非孤儿药在临床试验特征和临床开发成本方面的差异。
我们发现，每批准一种孤儿药的自费临床成本为 1.66 亿美元，每批准一种非孤儿药的临床成本为 2.91 亿美元（2013 年美元）。每批准一种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。当仅关注新分子实体时，我们发现，每批准一种孤儿药的资本化临床成本是非孤儿药的一半。
需要进行更多的讨论，以更好地协调应将哪些成本因素纳入药品研发成本。
