RTFCCR and Anticancer Fund (ACF) conducted a joint on-patent drug repurposing landscape analysis to identify the unmet need for philanthropic support. Many rare and ultra-rare cancer types remain without a treatment option. This high unmet need could be addressed by drug repurposing. Indeed, RTFCCR receives many requests from the medical community to support clinical research in this space.
On-patent drug-repurposing allows a possibility to find a new therapeutic use for an existing drug through leveraging knowledge previously generated on its safety and effectiveness. In this case, the drug development process is less costly and quicker than the traditional bench to bed-side cycle.
Typically, pharmaceutical companies are not interested in funding on-patent drug repurposing clinical trials. For many companies, the trajectory of development for a drug is decided even before the first approval and therefore the development of repurposing opportunities outside of those plans is often not supported. Companies may also judge the likelihood of competitor products becoming available when the drug is near the end of the exclusivity period, and the possibility that other drugs in the same class might be used in the new indication and therefore reduce the chance of a positive return on investment from their repurposing assets.
Therefore, philanthropic funds are needed to close this gap. RTFCCR decided to consider applications in this space on patient population with high unmet needs. Proposals will be assessed case-by-case taking into consideration whether the trial has the potential to establish new standards of care.
The data generated during the trials most likely will be used to support off-label use (evidence for clinicians to prescribe in a new indication) or in a less likely scenario for label extension (if the company is willing to file) – on-label use (see figure 1).
RTFCCR believes that the impact it can create, in order to improve patients outcomes, can be highly increased from new treatment options generated through this approach.