It is imperative to explore all viable avenues in pursuing innovative, effective therapies for patients with rare diseases and other unmet medical needs. One such promising avenue is the rapid and cost-effective repurposing of already existing and marketed drugs and drug combinations for new indications. These therapies often offer lower costs, speed, and reduced risks than newly patented drugs.
However, even these new therapies require investment and assurance of a fair and reasonable return on investment. This is currently not given due to unwillingness to reward these investments beyond the original reimbursement of medication costs for the previous indications. Thus, these innovations will not happen.
To harness the massive potential of drug repurposing, the REPO4EU platform and consortium -with the support of the EU- propose the establishment of a Special Interest Group (SIG) to advocate for a Guideline from the European Commission setting out best practices for health-technology agencies across different countries to reimburse repurposed drugs at a price that ensures a fair and reasonable return on investment to enable the final stages of development and the necessary investments.