Orphan drugs are specialized medicines designed to treat extremely rare diseases that affect only a small number of people. They are called orphans because, earlier, these diseases were ignored since making medicines for so few patients was not profitable.
Some Examples are:
Ivacaftor – for cystic fibrosis
Alglucerase – for Gaucher’s disease
Asfotase alfa – for hypophosphatasia (rare bone disorder)
Even though orphan drugs are developed for a small number of people with rare diseases, they are still very important. Each life is valuable, and developing vaccines as part of orphan drug initiatives can save lives. While there may be a profit motive, the significance of saving lives should always be the priority.
Orphan drugs are extremely important because they could the last hope of patients suffering from rare diseases.
Many countries encourage the development of orphan drugs despite small patient populations governments provide incentives such as:
1)Market exclusivity(7–10 years in the U.S.)
2)Tax credits and grants for research and development.
3)Accelerated or flexible regulatory approval processes.