Financing translation: Analysis of the NCATS rare-diseases portfolio

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 timeline...

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Main Authors: Fagnan, David E., Yang, N. Nora, McKew, John C., Lo, Andrew W
Other Authors: Massachusetts Institute of Technology. Computer Science and Artificial Intelligence Laboratory
Format: Article
Language:en_US
Published: American Association for the Advancement of Science (AAAS) 2015
Online Access:http://hdl.handle.net/1721.1/99456
https://orcid.org/0000-0003-2944-7773
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author Fagnan, David E.
Yang, N. Nora
McKew, John C.
Lo, Andrew W
author2 Massachusetts Institute of Technology. Computer Science and Artificial Intelligence Laboratory
author_facet Massachusetts Institute of Technology. Computer Science and Artificial Intelligence Laboratory
Fagnan, David E.
Yang, N. Nora
McKew, John C.
Lo, Andrew W
author_sort Fagnan, David E.
collection MIT
description 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.
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spelling mit-1721.1/994562023-03-01T02:14:40Z Financing translation: Analysis of the NCATS rare-diseases portfolio Fagnan, David E. Yang, N. Nora McKew, John C. Lo, Andrew W Massachusetts Institute of Technology. Computer Science and Artificial Intelligence Laboratory Massachusetts Institute of Technology. Department of Electrical Engineering and Computer Science Massachusetts Institute of Technology. Operations Research Center Sloan School of Management Lo, Andrew W. Fagnan, David E. Lo, Andrew W. 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. MIT Laboratory for Financial Engineering National Institutes of Health (U.S.). Intramural Research Program National Institutes of Health (U.S.). National Center for Advancing Translational Sciences 2015-10-26T16:46:38Z 2015-10-26T16:46:38Z 2015-02 Article http://purl.org/eprint/type/JournalArticle 1946-6234 1946-6242 http://hdl.handle.net/1721.1/99456 Fagnan, D. E., N. N. Yang, J. C. McKew, and A. W. Lo. “Financing Translation: Analysis of the NCATS Rare-Diseases Portfolio.” Science Translational Medicine 7, no. 276 (February 25, 2015): 276ps3–276ps3. https://orcid.org/0000-0003-2944-7773 en_US http://dx.doi.org/10.1126/scitranslmed.aaa2360 Science Translational Medicine Creative Commons Attribution-Noncommercial-Share Alike http://creativecommons.org/licenses/by-nc-sa/4.0/ application/octet-stream American Association for the Advancement of Science (AAAS) Prof. Lo via Shikha Sharma
spellingShingle Fagnan, David E.
Yang, N. Nora
McKew, John C.
Lo, Andrew W
Financing translation: Analysis of the NCATS rare-diseases portfolio
title Financing translation: Analysis of the NCATS rare-diseases portfolio
title_full Financing translation: Analysis of the NCATS rare-diseases portfolio
title_fullStr Financing translation: Analysis of the NCATS rare-diseases portfolio
title_full_unstemmed Financing translation: Analysis of the NCATS rare-diseases portfolio
title_short Financing translation: Analysis of the NCATS rare-diseases portfolio
title_sort financing translation analysis of the ncats rare diseases portfolio
url http://hdl.handle.net/1721.1/99456
https://orcid.org/0000-0003-2944-7773
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