How To Overcome Headwinds In Securing Funding For Rare Disease Research

By Giacomo Chiesi

In recent months drug developers have seen, and many have experienced, significant headwinds in securing funding, as evidenced by workforce layoffs as well as challenges in the ability of publicly listed biotech companies to raise money or for privately listed biotech companies to initiate a public offering.

The headwinds are possibly even stronger for drug developers in rare and orphan diseases, given they target very small markets and often must conduct expensive and sometimes lengthy clinical trials with complex manufacturing processes. VCs and other investors typically prioritize assets that target large patient populations and have broad applicability, given the higher prevalence might present more attractive “exits” or clearer liquidity events. As a result, many potential rare disease treatments languish due to lack of funding.