Off-Patent Drug Repurposing

Finding a new therapeutic use for an existing drug seems like a simple way to get more treatments to more patients more quickly. However, off-patent drugs generally have little potential for financial return on the investment made to repurpose the drug, particularly if generic forms of the drug are already on the market. There are a number of non-scientific challenges that stand in the way of testing scientific hypotheses for new uses of off-patent drugs. And an overarching challenge is that no single entity owns the issues. In December 2019, the National Center for Advancing Translational Sciences (NCATS), the U.S. Food and Drug Administration (FDA), and the Reagan-Udall Foundation for the FDA (RUF) held a workshop to discuss research and regulatory challenges of off-patent drug repurposing and formulate actionable solutions. Participants of the workshop included patients, philanthropic organizations, regulators, payors, healthcare economists, the U.S. Department of Veteran's Affairs (VA), and the National Institutes of Health (NIH).