An orphan drug is an agent used to address rare diseases. Orphan drugs work to prevent, diagnose and treat conditions affecting less than 200,000 people in the U.S. The 1983 Orphan Drug Act created the term “orphan drug” to financially incentivize drug companies to develop effective and safe drugs for rare diseases.
Why are orphan drugs important to healthcare?
Orphan drugs are important because they prevent, diagnose and treat rare diseases that researchers often ignored in the past. Thus, orphan drugs lead to better health outcomes and quality of life for individuals living with rare diseases.