The promise of the EU's draft repurposing programme

Area of Science:

• Pharmaceutical innovation
• Drug repurposing
• Regulatory science

Background:

• Government-led drug repurposing programs are changing pharmaceutical innovation.
• A proposed European Union (EU) reform grants the European Medicines Agency (EMA) authority to approve new therapeutic indications for marketed drugs without marketing authorization holder consent.
• This authority is opposed by companies but its potential patient benefit is poorly understood.

Purpose of the Study:

• To empirically assess the potential of the EMA's new repurposing authority.
• To analyze the volume and types of evidence-based new uses for existing medicines.
• To evaluate the implications for neglected patient populations.

Main Methods:

• Analysis of 198 medicines over 12 years.
• Comparison of EMA-authorized labels with US Food and Drug Administration (FDA) approvals and off-label use references.
• Identification of additional therapeutic indications supported by clinical evidence.

Main Results:

• 67% of analyzed medicines had at least one additional evidence-based use, totaling 320 potential new indications.
• 39% of these new uses were for new diseases, and 61% were for new patient cohorts.
• A third of new patient cohort uses involved pediatric populations, highlighting a potential benefit for neglected groups.

Conclusions:

• The EMA's proposed authority could facilitate the approval of numerous evidence-based drug repurposing uses, especially those already approved by the FDA.
• The findings support the policy focus on repurposing for new patient cohorts, including pediatric populations.
• This regulatory power presents a significant opportunity to address unmet medical needs in neglected populations.