Rare Disease Drug Development Series: What Patients and Advocates Need to Know

This course, which is Part 3 in our series “Rare Disease Drug Development: What Patients and Advocates Need to Know,” will cover:

• Clinical trial endpoints and Clinical Outcome Assessments (COAs)
• Designing and conducting clinical trials
• Designing clinical trials for small populations
• Patient experience data

The focus of this series is on rare diseases and how patients and patient advocates can play a significant role in rare disease drug development.

The course is available free online and can be accessed on mobile. It has been created by the National Organization for Rare Disorders (NORD) in collaboration with FDA’s Center for Drug Evaluation and Research (CDER) and the Critical Path Institute. Funding was made available through the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP), a collaborative agreement with the Critical Path Institute funded by FDA.