Abstract

EMBO Reports (2019) e48173 Philanthropy is a bright light in a dim reality of medical research and drug development. Only 1 in 10,000 academic discoveries make their way into a new therapy or diagnostics that helps patients. Those few discoveries that are taken up by biotech or pharma companies take 15 years and over US$1 billion to bring a new product to the market—if they ever make it that far. Moreover, there are between 7,000 and 9,600 known rare diseases, for which there are 773 FDA‐approved orphan drugs as of 2019 (https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm). These data are primarily US‐centric, but they represent the challenges faced around the world. > Time and again, philanthropy has shown to wield a profound and outsized impact because it is not subject to the same constraints as other sources of capital. Philanthropy is a relatively small player in the medical research ecosystem. It accounts for < 3% of all of US funding for R&D (https://www.researchamerica.org/sites/default/files/Policy_Advocacy/2013-2017InvestmentReportFall2018.pdf), which pales in comparison with the billions of dollars spent by government entities, such as the NIH, and the biopharma industry. Yet, underdog as it may be, it can still punch well above its weight. Time and again, philanthropy has shown to wield a profound and outsized impact because it is not subject to the same constraints as other sources of capital. Michael Milken, a medical philanthropist and Chairman of the Milken Institute, said “Philanthropy is far more than just writing checks. It takes an entrepreneurial approach that seeks out best practices and empowers people to change the world” (https://givingpledge.org/Pledger.aspx?id=245). When philanthropists and foundations fully embrace the unique attributes of their capital and use it strategically to fill key funding gaps, incentivize new behaviors, and innovate the medical research system, we see positive impacts for science and for patients. ### Philanthropy primed for long‐term growth The United States is in a second …

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