Overcoming barriers to off-patent drug repurposing: a lifecycle-based policy solutions

Area of Science:

• Pharmaceutical Sciences
• Health Policy
• Drug Development

Background:

• Drug repurposing offers a cost-effective approach to discovering new therapeutic uses for existing medicines.
• Off-patent drugs lack sufficient private investment incentives, and regulatory barriers hinder academic and non-profit sponsors.
• Prescribing by active ingredient presents a significant obstacle in drug repurposing initiatives.

Purpose of the Study:

• To identify key challenges in the lifecycle of off-patent drug repurposing.
• To explore potential policy solutions for facilitating drug repurposing.
• To assess the current landscape of drug repurposing for unmet health needs.

Main Methods:

• A structured literature review was conducted.
• A questionnaire was administered to 25 drug repurposing experts.
• The study analyzed challenges and solutions across the full drug repurposing lifecycle.

Main Results:

• Public funding is available for early-stage research, but support for confirmatory trials is limited.
• Successful drug authorization rates are below 30% due to limited regulatory focus from sponsors.
• Policy solutions include prioritizing rare diseases, increasing government investment, and fostering public-private partnerships.
• Indication-based differentiation and enhanced dispensing controls may address pharmacy substitution challenges.

Conclusions:

• Off-patent drug repurposing is hampered by persistent financial and regulatory barriers.
• Governmental engagement and support for academics and non-profits are essential.
• Despite recent progress, structural and political obstacles impede widespread implementation.