Rare neurological diseases present unique challenges for clinical development, where progress is often limited not only by scientific complexity, but by gaps in meaningful, high-quality data. In this episode of Cortex Conversations, experts explore how aligning regulatory expectations with real-world patient and caregiver needs can help overcome these barriers.

The discussion examines the critical role of natural history data in supporting external control arms and alternative trial designs, while also addressing the burden traditional site-based trials can place on patients—particularly in pediatric and neurodegenerative conditions. Participants share perspectives on emerging hybrid and decentralized approaches, the growing value of patient-driven data, and the collaborative efforts required to build natural history datasets that are both scientifically robust and practical for rare disease communities.

Together, the conversation highlights a path forward for advancing research in rare neurological diseases through innovation, partnership, and patient-centered trial design.

Host: 
Amber Burg, Therapeutic Delivery Head of Neuroscience and Ophthalmology at Fortrea 

Participants 
Louise Kearney, Executive Director of Cell and Gene Therapy at Fortrea

Hannah Simonds, Director of Patient Recruitment and Engagement at Fortrea

Karmen Trzupek, Senior Director of Scientific Programs, Global Genes