Accelerating Insights for Rare Genetic Disorders

This case study highlights how IDR supported a gene therapy and biotechnology company in addressing the unique challenges of Canavan disease and leukodystrophies through expert recruitment and actionable insights.

Overview

A leading gene therapy and biotechnology company wanted to deepen its understanding of the current state of knowledge and practice in treating Canavan disease and leukodystrophies, with a special focus on pediatric cases. Recognizing the rarity of these neurodegenerative disorders, they turned to IDR for support in engaging niche experts and participants.

IDR was commissioned to recruit two key groups: decision-makers responsible for reimbursement and approval of gene therapies (including participants in Pharmacy and Therapeutics Committees) and physicians and medical experts managing pediatric leukodystrophy cases at centers of excellence.

What We Did

• Custom Recruitment: Mobilized our global network to identify and recruit specialists, including neurologists, geneticists, and caregivers with expertise in rare genetic disorders.
• Tailored Screening: Designed detailed screening processes to ensure participants had the precise knowledge and experience needed for the study’s objectives.
• Rapid Delivery: Delivered the niche experts swiftly, within just two weeks, meeting tight research deadlines.
• Balanced Composition: Ensured a diverse mix of healthcare payers and medical practitioners to provide a well-rounded perspective.

Outcome

By working with IDR, the client gained actionable insights into the challenges and opportunities in diagnosing and treating Canavan disease and leukodystrophies. These insights informed their strategy for advancing diagnostic tools and developing innovative treatment modalities, particularly for pediatric cases.

The carefully selected experts provided profound knowledge and practical perspectives, enabling the client to build a strong foundation for future clinical research and therapeutic innovation.