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Financing translation: analysis of the NCATS rare-diseases portfolio.
Sci Transl Med
February 2015
Operations Research Center, Massachusetts Institute of Technology (MIT), Cambridge, MA 02142, USA. Sloan School of Management and Laboratory for Financial Engineering, MIT, Cambridge, MA 02142, USA. Computer Science and Artificial Intelligence Laboratory and Department of Electrical Engineering and Computer Science, MIT, Cambridge, MA 02139, USA. AlphaSimplex Group, LLC, Cambridge, MA 02142, USA.
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.
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