Massachusetts Institute of Technology (MIT) Sloan School of Management and Laboratory for Financial Engineering, Cambridge, MA 02142, USA. MIT Computer Science and Artificial Intelligence Laboratory and Department of Electrical Engineering and Computer Science, Cambridge, MA 02142, USA. AlphaSimplex Group, Cambridge, MA 02142, USA.
Early Clinical Development, Genentech, South San Francisco, CA 94080, USA.
Sci Transl Med. 2014 Jun 18;6(241):241cm5. doi: 10.1126/scitranslmed.3008228.
As the prevalence of Alzheimer's disease (AD) grows, so do the costs it imposes on society. Scientific, clinical, and financial interests have focused current drug discovery efforts largely on the single biological pathway that leads to amyloid deposition. This effort has resulted in slow progress and disappointing outcomes. Here, we describe a "portfolio approach" in which multiple distinct drug development projects are undertaken simultaneously. Although a greater upfront investment is required, the probability of at least one success should be higher with "multiple shots on goal," increasing the efficiency of this undertaking. However, our portfolio simulations show that the risk-adjusted return on investment of parallel discovery is insufficient to attract private-sector funding. Nevertheless, the future cost savings of an effective AD therapy to Medicare and Medicaid far exceed this investment, suggesting that government funding is both essential and financially beneficial.
随着阿尔茨海默病（AD）的患病率不断上升，其给社会带来的成本也在不断增加。科学、临床和财务利益主要集中在导致淀粉样蛋白沉积的单一生物学途径上，从而推动了当前的药物发现工作。这一努力进展缓慢，结果令人失望。在这里，我们描述了一种“投资组合方法”，其中同时进行多个不同的药物开发项目。虽然需要更多的前期投资，但通过“多个目标射击”，至少有一个成功的概率应该更高，从而提高这项工作的效率。然而，我们的投资组合模拟表明，平行发现的风险调整投资回报率不足以吸引私营部门的资金。尽管如此，一种有效的 AD 疗法对医疗保险和医疗补助的未来成本节约远远超过了这项投资，这表明政府资金是必不可少的，而且在财务上也是有益的。
