Rare Disease Drug Repurposing.

Area of Science:

• Drug Repurposing
• Rare Diseases
• Nonprofit Organizations

Background:

• Over 9500 rare diseases lack US Food and Drug Administration-approved therapies, necessitating innovative treatment strategies.
• Drug repurposing offers a potentially faster and more cost-effective alternative to novel drug development, but faces significant hurdles.
• Rare disease nonprofit organizations (RDNPs) have historically led repurposing efforts, yet systematic data on their successes are limited.

Purpose of the Study:

• To characterize the current landscape of rare disease drug repurposing initiatives.
• To identify key factors contributing to successful drug repurposing outcomes.
• To provide thematic insights for optimizing rare disease drug repurposing through the ROADMAP Project web tool.

Main Methods:

• A qualitative, mixed-methods study involving US-based RDNPs and stakeholders.
• Data collection included a national survey and semistructured interviews with RDNP leaders (September 2021 - January 2022).
• Analysis employed random forest models to identify factors influencing repurposing project stage and qualitative thematic synthesis.

Main Results:

• Survey responses from 147 RDNPs indicated 42% were involved in repurposing, with 94 drugs in various stages.
• Successful outcomes were significantly associated with nonprofit-supported patient recruitment (P < .001) and nonfinancial research support (P = .02).
• A 5-stage repurposing framework was developed, highlighting critical steps from identification to clinical practice optimization.

Conclusions:

• RDNPs play a crucial role in advancing rare disease drug repurposing.
• Optimizing systematic repurposing requires strategic support for patient recruitment and research.
• The study offers actionable insights for RDNPs, collaborators, and policymakers to facilitate data-driven drug repurposing.