The term "orphan drug" refers to what?

The term "orphan drug" specifically refers to medications developed to treat rare diseases, which are often overlooked due to the limited market potential. The designation is significant in pharmacology and public health because it highlights the need to support the development of treatments for conditions that affect a small number of patients, typically defined as those affecting fewer than 200,000 individuals in the United States.

Regulatory bodies, such as the FDA, offer incentives for the development of orphan drugs, including tax credits, grant funding, and extended market exclusivity, to encourage pharmaceutical companies to invest in research and development for these rare conditions. This is crucial as many rare diseases may not have any approved treatment options, leaving patients without effective therapies.

Understanding the context of orphan drugs emphasizes the importance of addressing healthcare needs across the spectrum of disease prevalence, recognizing that not all conditions receive equal attention in pharmaceutical innovation.