TBD: Medicines repurposing: moving from an emergency solution to a sustainable strategy to address unmet medical need
Martin De Kort
Senio Scientific Program Manager
EATRIS ERIC Netherlands
•Capture opportunities and challenges for the repurposing of well-established medicines in the EU
•Assess the STAMP repurposing pilot experience, looking at how the lessons learnt through the pilot can lead to optimization of the EU regulatory framework
•Identify the steps needed to create an EU ecosystem that bolsters medicines repurposing
Drug repurposing matured in the last decades as a strategy to identify new indications for well-established molecules to address unmet medical needs. The Covid-19 pandemic highlights its importance as a potential strategy to deliver safe, effective and affordable medicines with significantly shorter timelines compared to traditional R&D. Repurposing can be applied across a wide range of diseases, including those where other R&D approaches have failed to provide solutions for patients (e.g rare tumors or neurological diseases). This opportunity was recognized by policymakers in the Pharmaceutical Strategy for Europe and with the STAMP pilot project, which will inform possible regulatory action for the promotion of drug repurposing.
Despite promising policy steps, increasing funding and research attention, systemic inefficiencies for repurposing in the EU remain: 1) a fragmented and siloed R&D environment without an identifiable value chain for patient-centric repurposing; 2) heterogeneous quality and access to reliable computational tools using standardized datasets; 3); health technology assessment, pricing and reimbursement barriers; 4) non-alignment of the regulatory landscape with repurposing needs.
Thus, more is needed to promote specific evidence generation efforts, address the issue of off-label use of repurposed medicines and build a policy environment that facilitates and rewards continuous innovation on well-established molecules.
This session will bring together regulators, patient advocates, policy makers and industry representatives to explore how to advance the European repurposing ecosystem. Panelists will build on the lessons learnt to date from the STAMP pilot to assess how the regulatory landscape should evolve and identify sustainable strategies to bolster repurposing in the EU. A particular focus will be on enablers of long-term synergies, such as the recently-launched EU Horizon funded REMEDI4ALL platform.