Related Experiment Video
Updated: May 5, 2026

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Financing drug discovery for orphan diseases
David E Fagnan1, Austin A Gromatzky2, Roger M Stein3
1MIT Laboratory for Financial Engineering, Sloan School of Management, Cambridge, MA, USA; MIT Operations Research Center, Cambridge, MA, USA.
Abstract:
Recently proposed 'megafund' financing methods for funding translational medicine and drug development require billions of dollars in capital per megafund to de-risk the drug discovery process enough to issue long-term bonds. Here, we demonstrate that the same financing methods can be applied to orphan drug development but, because of the unique nature of orphan diseases and therapeutics (lower development costs, faster FDA approval times, lower failure rates and lower correlation of failures among disease targets) the amount of capital needed to de-risk such portfolios is much lower in this field. Numerical simulations suggest that an orphan disease megafund of only US$575 million can yield double-digit expected rates of return with only 10-20 projects in the portfolio.
Related Concept Videos
Prescription, Nonprescription and Orphan Drugs
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
Drug Discovery: Overview
Pharmacogenomics: Identification of New Drug Targets
Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions
Preclinical Development: Overview
Clinical Trials: Overview

