Barriers and Solutions Across Drug Repurposing Archetypes

In collaboration with Every Cure we present the following white paper: Barriers and Solutions Across Drug Repurposing Archetypes.

Millions of patients worldwide live with conditions that have no approved treatments or face barriers to accessing existing therapies. Traditional drug development is slow, costly, and commercially driven, leaving many potential therapies unexplored. Drug repurposing, identifying new uses for existing drugs beyond their original approved indications, is a promising approach that could bring treatments to millions of people around the world. But despite the vast potential of drug repurposing, it is often hindered by an array of challenges that result from a drug development ecosystem largely dominated by commercial drug developers.

Every Cure, a nonprofit on a mission to save and improve lives by repurposing drugs, categorizes drug repurposing opportunities into four archetypes based on their stage of evidence and adoption:

- Frontier explorer: Repurposing opportunity that shows promise based on mechanism, biological plausibility, or initial observational data, but requires further laboratory research before clinical evaluation.
- Clinical gem: Repurposing opportunity with sufficient preclinical and early clinical data to study in patients, but insufficient evidence to support recommending its use to physicians, requiring further clinical research to clarify efficacy and guide treatment.
- Unsung hero: Repurposing opportunity with clear and compelling clinical evidence to support its use, but limited adoption in practice, where targeted evidence generation and advocacy can help guide uptake.
- Known entity: A drug that is already widely used off-label for a particular condition, with substantial awareness and adoption among clinicians.

Each archetype presents distinct challenges on the path to patient use and impact. To fully unlock the potential of drug repurposing, cross-sector leaders must take coordinated action to align incentives, generate evidence, and expand access to life-saving therapies.