Shining light on the global network of rare diseases: Unveiling the 'rare' in not so rare

Orphan drugs developed to treat rare diseases represent a vital, yet often overlooked facet of modern healthcare. Rare diseases, affecting a small percentage of the population, pose significant challenges for individuals and healthcare systems. Despite their rarity, collectively, rare diseases impact millions worldwide. This review explores the interconnected realms of orphan drugs and rare diseases, defining their significance, the challenges faced by patients, and the pivotal role of orphan drugs in addressing unmet medical needs. Through legislative incentives and targeted research efforts, orphan drugs offer hope to individuals with rare diseases, providing tailored treatments and improving quality of life. Understanding and addressing the needs of this patient population are crucial steps toward achieving health equity and advancing healthcare innovation.