Robertson Clinical and Translational Cell Therapy Program, Duke Translational Medicine Institute, Duke University, Durham, North Carolina, USA.
Health Aff (Millwood). 2012 Nov;31(11):2528-35. doi: 10.1377/hlthaff.2012.0235.
The 1983 Orphan Drug Act created incentives for the development of orphan drugs. Despite its successes, including a substantial increase in new drugs, approved orphan drugs still treat fewer than 5 percent of registered rare diseases. In addition, concerns have arisen about the high prices of many of these therapies, which can cost hundreds of thousands of dollars per patient each year. In this article, we propose a new "grant-and-access pathway," in which drug developers could opt to compete for federal grants to subsidize the costs of clinical testing. In return for the grant funding, companies would no longer claim orphan drug tax credits and would agree to price caps for marketed products based on the duration and costs associated with drug development, expected market size, and target rate of return. We identify scenarios in which such a policy could provide a net benefit to society.
1983 年《孤儿药法案》为孤儿药的开发创造了激励机制。尽管该法案取得了成功，包括新药数量的大幅增加，但批准的孤儿药仍不到登记在册的罕见病的 5%。此外，人们对许多这类疗法的高昂价格表示担忧，这些疗法每年每个患者可能要花费数十万美元。在本文中，我们提出了一种新的“授权和准入途径”，在此途径中，药物开发商可以选择竞争联邦拨款，以补贴临床试验的成本。作为获得拨款的回报，公司将不再申请孤儿药税收抵免，并将根据药物开发的持续时间和成本、预期市场规模以及目标回报率，同意对上市产品进行价格上限。我们确定了在这种情况下，该政策可能会给社会带来净收益的情景。
