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Megafund financing methods for funding early-stage translational research often require billions of dollars in capital to diversify idiosyncratic scientific and clinical risk enough to attract

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Megafund financing methods for funding early-stage translational research often require billions of dollars in capital to diversify idiosyncratic scientific and clinical risk enough to attract private- sector capital. In this problem set we apply this financing method to orphan drug development, where development costs, failure rates, and correlations are low, and therefore the amount of capital required to de-risk these portfolios is much lower. Consider a portfolio of 8 preclinical orphan drug compounds each initially acquired and developed using US$25 million of capital. Assume this capital is used to first purchase and then develop the compounds, with excess capital not currently deployed earning a 0% return in cash. For simplicity, also assume that compounds are sold once they (successfully) complete the preclinical phase. We will perform a statistical analysis to estimate the expected return of this investment over the initial preclinical research phase. Problem 2 0.0/1.0 point (graded) Using the estimates for clinical trial costs, success rates, and duration in the table below, estimate the expected value of an orphan drug at the beginning of the following phases. Assume that the effective annual cost of capital for preclincal research, Phase 1, Phase 2, Phase 3, and NDA review are 30%, 30%, 30%, 25%, and 15%, respectively, and that clinical trial costs are incurred at the beginning of each phase. (Note: Your answer should be expressed in units of millions of dollars.) Dui Phase Cost (US$ million) Probability of Success (yo Preclinical 5 69% 1 Phase 1 5 84% 1 Phase 2 8 53% 2 Phase 3 43 74% 2 NDA 96% C NDA: $ million Phase III: $ $ million Phase II: $ million . Phase 1: $ million Preclinical: $ million Megafund financing methods for funding early-stage translational research often require billions of dollars in capital to diversify idiosyncratic scientific and clinical risk enough to attract private- sector capital. In this problem set we apply this financing method to orphan drug development, where development costs, failure rates, and correlations are low, and therefore the amount of capital required to de-risk these portfolios is much lower. Consider a portfolio of 8 preclinical orphan drug compounds each initially acquired and developed using US$25 million of capital. Assume this capital is used to first purchase and then develop the compounds, with excess capital not currently deployed earning a 0% return in cash. For simplicity, also assume that compounds are sold once they (successfully) complete the preclinical phase. We will perform a statistical analysis to estimate the expected return of this investment over the initial preclinical research phase. Problem 2 0.0/1.0 point (graded) Using the estimates for clinical trial costs, success rates, and duration in the table below, estimate the expected value of an orphan drug at the beginning of the following phases. Assume that the effective annual cost of capital for preclincal research, Phase 1, Phase 2, Phase 3, and NDA review are 30%, 30%, 30%, 25%, and 15%, respectively, and that clinical trial costs are incurred at the beginning of each phase. (Note: Your answer should be expressed in units of millions of dollars.) Dui Phase Cost (US$ million) Probability of Success (yo Preclinical 5 69% 1 Phase 1 5 84% 1 Phase 2 8 53% 2 Phase 3 43 74% 2 NDA 96% C NDA: $ million Phase III: $ $ million Phase II: $ million . Phase 1: $ million Preclinical: $ million

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