Overview

Some of the brief’s key messages are:

Repurposing is a strategy to identify new uses for approved or investigational medicines outside the scope of their original medical indication.

While delivering innovation (new treatments that resolve unaddressed health needs), repurposing also offers several advantages over de novo (from scratch) development, such as lower costs of development, lower risk of failure and reduced time frame to registration.

Across almost all cancer types, many products are already commonly used off-label – in particular, for patients who have no alternative options. Off-label use means that patients receive a medicine without a clearly established benefit–risk ratio.

Non-commercial repurposing of off-patent medicines for cancer treatment has the potential of addressing currently unmet needs in a cost-effective way, especially in areas that are not attractive for the industry, such as rare cancers. Collectively, rare cancers account for around 22% of new cases in Europe.

While repurposing previously relied on an ad hoc discovery process, it has more recently evolved to rely on implementation of organized, systematic, data-driven approaches to identify suitable candidates. In most cases, these approaches integrate computational assistance. Big Data and artificial intelligence are increasingly used for this purpose.