Rare and orphan diseases: Challenges and opportunities

Rare and orphan diseases, also known as rare disorders, encompass a diverse array of prerequisites which collectively impact a substantial number of people worldwide. Despite their low individual prevalence, these diseases pose multifaceted challenges for patients, health stakeholders, and pharmaceutical companies. This comprehensive review article delves into the intricate landscape of rare and orphan diseases, exploring their definition, epidemiology, diagnostic complexities, treatment options, and the ongoing efforts to change the unique hurdles associated with these conditions. By shedding light on this often-overlooked area of healthcare, we aim to bring the issue to light and encourage widespread understanding, and ultimately pave the way for improved outcomes and quality of life for those affected by these rare disorders.