Strategies to Advance Drug Repurposing for Rare Diseases

While the number of novel treatments for rare diseases has grown substantially over the past few decades, many rare diseases still lack FDA-approved therapies. Scientists and physicians have long recognized a complementary source of treatments that might prove useful for patients with rare diseases: drugs already in wide use. Drug repurposing, which involves identifying different therapeutic uses for already-existing drugs, offers some potential efficiencies in the drug development process. For example, by leveraging established safety data, repurposed drugs may allow investigators more certainty in knowing what to expect when designing clinical trials. Despite its potential, however, systematic drug repurposing remains constrained by lack of attention from traditional for-profit pharmaceutical enterprises. Nijim et al. show that successful repurposing is possible when non-profits are engaged due to their ability to leverage patient advocacy networks, registries, and direct outreach, which can in turn accelerate trial enrollment and help ensure studies reflect the characteristics of the broader patient populations in routine care settings.