Rare diseases occur globally at every stage of life. The drugs which are used to diagnose or treat rare medical conditions such as cystic fibrosis, multiple sclerosis, narcolepsy etc. are called Orphan drugs. These drugs are called orphan drugs because pharmaceutical companies do not put enough effort because the amount of money invested by the pharmaceutical company to develop the drug would not be recovered by the sale of the drug. The primary purpose of orphan drugs is to fill a critical void in the treatment landscape by targeting rare diseases that have been historically neglected by pharmaceutical development. While each orphan disease affects only a small number of patients, collectively they impact a substantial portion of the population. Orphan drugs are engineered to provide effective treatment options where none may have existed before, offering hope to individuals and families grappling with the challenges of rare diseases. Around 464 drugs only from oncology received this designation till 2020. Around 5644 drugs were designated orphan status at US-FDA till July 2023.
