CURE ID: A Platform to Collect Real-World Treatment Data for Drug Repurposing in Rare Genetic Disorders

Area of Science:

• Medical Science
• Pharmacology
• Genetics

Background:

• Rare diseases impact millions, yet approved treatments are scarce, hindering drug development.
• Off-label drug use is common in rare genetic disorders but data is often inaccessible for research.
• Drug repurposing presents a viable strategy to discover novel treatments for rare conditions.

Purpose of the Study:

• To introduce CURE ID, a novel treatment registry for rare diseases.
• To demonstrate how real-world data can accelerate the identification of repurposed drugs for rare conditions.
• To highlight the importance of community adoption for the success of CURE ID.

Main Methods:

• CURE ID collects real-world treatment data from healthcare providers, patients, and care partners.
• Data is gathered in a consistent format to enable aggregation and analysis.
• The platform aims to generate hypotheses for targeted research on repurposed drugs.

Main Results:

• The registry aggregates treatment experiences to identify potential drug repurposing candidates.
• Captured data can inform the evaluation of drug safety and efficacy for new rare disease indications.
• Successful hypothesis generation relies on widespread community reporting to CURE ID.

Conclusions:

• CURE ID serves as a valuable public resource for rare disease treatment research.
• Leveraging real-world data through registries like CURE ID can expedite the drug repurposing process.
• Increased community engagement is crucial for advancing treatment options for rare diseases.