Engaging Stakeholders in Rare Disease Clinical Trials

Rare diseases affect millions worldwide, yet developing treatments remains a challenge.

A new position paper from ISCTM explores how feedback from patients, caregivers, clinicians, and advocacy groups can transform the drug development process.

Learn about best practices to overcome obstacles like disease understanding, endpoint development, and competing research priorities.

Read the full article to discover how collaborative efforts are shaping the future of rare disease therapeutics.

This article was published in Innovations in Clinical Neuroscience (January-March 2024). Authors include Gahan J. Pandina, Joan Busner, Lucas Kempf, Joan Fallon, and other members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development.

Disclaimer: This position paper reflects the views of the authors as members of the ISCTM Working Group and should not be understood or quoted as being made on behalf of or reflecting the position of the agencies or organizations with which the authors are employed/affiliated.