How are orphan drugs typically classified?

Orphan drugs are specifically classified by their indication for rare diseases. These drugs are developed to treat conditions that affect a small number of patients, often referred to as orphan diseases, which can be under 200,000 cases in the United States. This classification is based on the unique challenge of addressing medical needs that are not profitable for the pharmaceutical industry due to the limited market size.

By focusing on their indication for rare diseases, orphan drugs receive special designations and incentives from regulatory authorities to encourage their development. This can include benefits such as tax credits, grant funding, and extended market exclusivity. The emphasis on rare disease indications underscores the importance of providing treatment options for conditions that may otherwise go unaddressed due to lack of financial incentive for drug development.

Other classifications, like active ingredients, market availability, or development costs, do not encapsulate the core mission behind orphan drug designation, which is inherently tied to their role in addressing the therapeutic needs related to rare diseases.