Financing translation: Analysis of the NCATS rare-diseases portfolio
Financing translation: Analysis of the NCATS rare-diseases portfolio
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DOI：
10.1126/scitranslmed.aaa2360
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发表时间：
2015-02-25
影响因子：
17.1
通讯作者：
Lo, Andrew W.
中科院分区：
文献类型：
--
作者：
Fagnan, David E.;Yang, N. Nora;Lo, Andrew W.
国家转化科学推进中心（NCATS）罕见病治疗开发项目的投资组合包括28个在临床前阶段启动的研究项目。历史数据显示，相对于文献中通常引用的早期转化医学研究和开发（R&D）的行业平均水平，NCATS项目的成本显著降低，成功率更高，但临床前时间更长。在这里，我们评估了投资罕见病治疗组合的潜在风险和回报。使用“巨型基金”融资结构，NCATS数据和行业专家小组的估值估计，我们模拟了一个假设的巨型基金，其中高级和次级债务分别收益率为5%和8%。模拟的预期股本回报率为14.7%，对应的修正内部回报率为21.6%。这些回报和私营部门资助的可能性可以通过来自慈善机构、患者倡导团体和政府机构的第三方资金担保来提高。
The portfolio of the National Center for Advancing Translational Sciences (NCATS) rare-diseases therapeutic development program comprises 28 research projects initiated at the preclinical stage. Historical data reveal substantially lower costs and higher success rates but longer preclinical timelines for the NCATS projects relative to the industry averages for early-stage translational medical research and development (R&D) typically cited in literature. Here, we evaluate the potential risks and rewards of investing in a portfolio of rare-disease therapeutics. Using a "megafund" financing structure, NCATS data, and valuation estimates from a panel of industry experts, we simulate a hypothetical megafund in which senior and junior debt yielded 5 and 8%, respectively. The simulated expected return to equity was 14.7%, corresponding to a modified internal rate of return of 21.6%. These returns and the likelihood of private-sector funding can be enhanced through third-party funding guarantees from philanthropies, patient advocacy groups, and government agencies.