RARE - Dr. Selena Freisens

Unlocking New Possibilities: The Power of Drug Repurposing for Rare Diseases

For this episode, Laura Hulley, Principal Consultant at SciPro had the pleasure of speaking with Dr. Selena Freisens, a seasoned physician with over 25 years of experience in the Pharma and Biotech industries, shared her insights on the transformative potential of drug repurposing for rare diseases (RDs). With decades of work in executive medical affairs and a passion for addressing unmet medical needs, Selena highlighted the value, challenges, and future of drug repurposing in a rapidly evolving healthcare landscape.

 

What is Drug Repurposing?

Drug repurposing, or repositioning, involves developing an existing drug for a new regulatory-approved indication. Simply put, it takes a drug already approved for one condition and applies it to another. This approach leverages existing knowledge about the drug’s properties, including its active ingredients, mechanisms of action, and safety profile.

The Value of Drug Repurposing

Drug repurposing offers several benefits that could significantly impact healthcare, particularly in the rare disease space:

• Faster and More Affordable Development: By utilizing existing data and reducing the time required for trials, repurposing can expedite the availability of cost-effective treatments.
• Crisis Response: During the COVID-19 pandemic, over 100 repurposed drugs entered clinical trials, with several receiving FDA approval. This demonstrates how quickly repurposing can address urgent medical needs.
• Unlocking Untapped Potential: Organizations like the World Health Organization’s anti-cancer fund have identified hundreds of medicines with potential for repurposing, showcasing the vast opportunities available.

 

Why Rare Diseases Need Drug Repurposing

Rare diseases face unique challenges, including limited understanding of disease mechanisms, small patient populations, and high treatment costs. Of the approximately 10,000 known rare diseases, only 5% have approved treatments. Developing new therapies is often prohibitively expensive and time-consuming.

Drug repurposing provides a viable alternative, offering hope to millions by accelerating access to effective treatments. Selena pointed to successful examples, such as:

• Propranolol: Originally approved for hypertension, later repurposed to treat infantile hemangioma.
• Fenfluramine: Once an appetite suppressant, it was later approved for treating Dravet syndrome and Lennox-Gastaut syndrome.

 

Challenges in Drug Repurposing

Despite its promise, drug repurposing faces significant obstacles:

• Commercial Disincentives: Without market protection for generic drugs with new indications, pharmaceutical companies are reluctant to invest.
• Lack of Resources in Academia: Academic researchers often lack the funding, time, and knowledge to pursue clinical trials.
• Systemic Barriers: National healthcare systems may prioritize the cheapest generic drugs, disincentivizing innovation.

Selena stressed the need for systematic strategies to identify and link existing drugs to new indications.

 

Advancing Drug Repurposing: Regulatory Support and Innovation

Recent regulatory initiatives aim to address these barriers. Key efforts include:

• EMA’s STAMP Project: Supports not-for-profit organisations and academic institutions in generating evidence for regulatory approval.
• CURE ID: An FDA-backed platform for real-world data collection.
• REMEDi4ALL and REPO4EU: EU-funded initiatives advancing systematic drug repurposing efforts.

Technological advancements, such as machine learning and artificial intelligence, are also revolutionising repurposing by predicting potential new uses for drugs based on biological, clinical, and chemical data. For example, the Every Cure LinkMap evaluates thousands of FDA-approved drugs against a vast array of diseases.

The Road Ahead

As patient advocacy grows and digital tools become more sophisticated, the future of drug repurposing looks promising. However, Selena emphasised the need for sustainable revenue models and policy changes to incentivise investment in this field.
Ultimately, drug repurposing is more than just finding new uses for existing medications—it’s a lifeline for patients with rare diseases. With collaboration among stakeholders, advancements in technology, and continued regulatory support, the healthcare industry can transform this potential into reality.

Drug repurposing represents an innovative, cost-effective pathway to address the unmet needs of rare disease patients worldwide. While challenges remain, the combined efforts of governments, researchers, and patient communities could soon make this approach a cornerstone of rare disease treatment.

Thank you Selena for taking the time to feature!