The Orphan Drug Act (ODA) of 1983 is a critical milestone in clinical research, especially for rare diseases affecting fewer than 200,000 people in the U.S. Prior to the ODA, only 38 orphan drugs were approved. Since its enactment, over 600 orphan drugs have been approved, indicating a significant shift in pharmaceutical research and development. This Act provides incentives such as tax credits, seven-year market exclusivity, and grant funding, encouraging companies to invest in orphan drug research. The impact is substantial: as of 2020, over 7,000 rare diseases have been identified, affecting an estimated 25-30 million Americans. Orphan drugs now represent over 20% of pharmaceutical market revenues, reflecting their growing importance. The ODA's influence extends beyond just numbers. By focusing on less common diseases, it has opened new avenues in medical research, often leading to broader applications in more common conditions. For instance, research on rare genetic disorders has enhanced understanding of genetics in general medicine, and the new field of Cell and Gene Therapy, CGT, is expanding into many indications. The Orphan Drug Act has not only provided hope and treatment options to millions with rare diseases but also advanced the overall field of medical research. More: https://en.wikipedia.org/wiki/Orphan_Drug_Act_of_1983 https://en.wikipedia.org/wiki/Orphan_drug #RareDiseases #CGT #ClinicalTrials #Healthcare Text modified from ChatGPT, Art by Dall-E.