Developing treatments for rare diseases involves unique obstacles due to the rarity and complexity of these conditions. To address these challenges, the FDA offers guidance on conducting natural history studies. These studies are essential for comprehending disease progression, identifying patient subgroups, discovering biomarkers, and refining clinical outcome assessments – all of which enhance the effectiveness and design of clinical trials.
Following the FDA's guidance ensures thorough data collection, precise disease modelling, and adherence to regulatory standards. In the report below, we've outlined the guidance for researchers and sponsors advancing drug development and delivering treatments for patients with rare diseases.
