It is a strategy to use existing drugs for new indications. It can reduce the cost and time of drug development and, in a short time, provide new treatment options for patients.
RTFCCR funds interventional clinical trials supporting off-patent drug repurposing, and in special cases (described below) also on-patent drug repurposing applications.
On-patent drug repurposing
RTFCCR and Anticancer Fund (ACF) conducted a joint on-patent drug repurposing landscape analysis to identify the field’s unmet need for philanthropic support.
Download the white paper on drug repurposing
Unfortunately, there are no treatment options for many rare and ultra-rare cancer types. However, this problem could be addressed by repurposing existing drugs into new indications and/or patient populations.
By leveraging existing knowledge about the safety and effectiveness of a known drug, on-patent drug-repurposing could find a new therapeutic use for existing treatments. The drug development process is usually cheaper and faster than the traditional bench-to-bedside cycle. Pharmaceutical companies are often not interested in funding on-patent drug repurposing clinical trials. For many companies, the development trajectory of a drug is decided before the first approval and the development of repurposing opportunities outside of those plans is typically not supported. When drugs are nearing the end of their exclusivity period, companies may consider the availability of competitor products and decide against repurposing their existing treatments, considering other drugs in the same class might become widely available.
Therefore, philanthropic funds are needed to close this gap. RTFCCR has decided to consider applications in this space on patient populations with high unmet needs. Proposals will be assessed case-by-case, taking into consideration the likelihood of the trial to establish new standards of care.
The data generated during the trials will likely 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. RTFCCR believes that the impact it can create to improve patient outcomes can be accelerated through the new treatment options generated through this approach.
On-patent drug repurposing LOI due diligence
Clinical trials on both, off-patent and on-patent drugs are eligible for Rising Tide Foundation for Clinical Cancer Research (RTFCCR) funding. Trial designs must include patient partners and demonstrate a truly patient-centered approach. Proposed trials must address potential benefits to patients enrolled in the study and include outcomes of patient-reported quality-of-life measures.
In addition to the thorough grant review process, on-patent drug project LOIs will be categorized according to their potential to establish new standards of care. The RTFCCR team will then utilize the decision trees listed below to select applications for the next steps.
High unmet need
- No treatment available
- Rare or ultra-rare population: incidence less than 1/10000 population (WHO guidelines)