Tag: Orphan drug act

Affecting an estimated one in ten Americans, there are more than 7,000 rare diseases, and for these patients, an orphan drug provides a critical, often life-saving, treatment option. Historically, the small patient populations for these conditions made drug development financially unviable, leading to a significant gap in treatment options.

In 2024, the global orphan drug market reached an estimated revenue of nearly $190 billion, illustrating the significant financial impact of these medications. This success raises the question: **What is the highest grossing orphan drug?** The answer is more complex than it appears, as many top sellers have expanded indications far beyond their initial orphan status.

Over 7,000 rare diseases collectively affect more than 30 million Americans, yet historically, most have had no approved treatment. Orphan drugs are specialized medications developed to treat, prevent, or diagnose these rare conditions, addressing a critical, unmet medical need.

According to a 2020 study, 108 branded drugs approved between 1983 and 2017 in the U.S. had multiple orphan indications, demonstrating that a drug can have multiple orphan drug designations. This practice of pursuing multiple indications for the same drug has significant implications for patients, pharmaceutical companies, and the broader healthcare system.