Factors to consider when designing clinical trials for repurposed drugs.

INTRODUCTION: Drug repurposing is the strategy of identifying novel therapeutic uses for existing, unapproved, or failed drugs distinct from their original indications. Given the unique nature of repurposing, clinical trials for repurposed agents warrant specific design considerations.

AREAS COVERED: This article covers specific factors to consider when designing clinical trials for repurposed agents. The specific factors identified are the drug's characteristics, its clinical context, the design and methodology of the repurposing trial, the quality and management of supporting data, and other ethical and organizational concerns specific to repurposing.

EXPERT OPINION: Drug repurposing is a natural approach to reduce costs and increase the efficiency of drug development, and such trials may offer ethical advantages in terms of participant risk and social value. In the modern landscape of clinical trials, numerous barriers to initiating repurposing efforts exist. Notably, these include challenges related to intellectual property, data availability, and the unexpected and widespread consequences of off-label use and drug access. Other barriers to safe and effective repurposing will continue to challenge trialists and stakeholders, especially regarding the reuse of data, communication, documentation, and other potential confounding factors not experienced in standard trials. Thus, careful ethical assessment of trial characteristics is necessary.