Orphan Drugs: A Lifeline for Rare Disease Patients
In the vast world of pharmaceuticals, orphan drugs have emerged as a powerful force in addressing the needs of patients with rare and often overlooked medical conditions. These specialized drugs are developed specifically to treat rare diseases, offering hope where few treatments exist. Despite their limited commercial appeal, orphan drugs are making a profound impact on global healthcare. What Are Orphan Drugs? Orphan drugs are pharmaceutical products intended for the diagnosis, prevention, or treatment of rare medical conditions, often referred to as orphan or rare diseases. These diseases affect a small percentage of the population—too few to justify the high cost of drug development under traditional market conditions. Globally, regulatory bodies define "rare" differently: - In the United States, a rare disease affects fewer than 200,000 individuals. - In the European Union, the threshold is 1 in 2,000 people. - In Japan, it's fewer than 50,000 patients. Given these s...