Case Study - Canavan Diseases and Leukodystrophies

Overview

A gene therapy/biotechnological company researched to gain a better understanding of the current state of knowledge and practice in the field of Canavan Diseases and leukodystrophies, with a special focus on pediatric cases.

We were commissioned to recruit on two scopes: Payer decision makers with an in-depth understanding of gene therapies and participation in P&T committees, and Physicians and Medical Experts managing pediatric leukodystrophy cases within leukodystrophy centers of excellence.

What we did

We successfully demonstrated our adeptness in efficiently sourcing and coordinating all five participants within a remarkably swift two-week timeframe.

This achievement was accomplished with a strategic emphasis on ensuring a well-balanced composition that encompassed both healthcare payers and medical practitioners, thereby enhancing the diversity and depth of expertise among the participants.

Outcome

The insights gleaned from this study hold paramount significance in the forthcoming development of advanced diagnostic tools and novel treatment modalities for Canavan disease.

The medical experts who generously shared their expertise and experiences during our interviews proved to be not only exceptionally cooperative but also profoundly insightful, contributing significantly to the foundation upon which future innovations in Canavan disease management will be built.

Experts engaged (sample)