Which of the following best describes an orphan drug?

Which of the following best describes an orphan drug?

• A. A drug used for common ailments
• B. A drug for rare diseases
• C. A drug with no market competition
• D. A drug with high annual sales

Answer: (B)

An orphan drug is defined as a medication specifically developed to treat a rare disease or condition, often affecting fewer than 200,000 individuals in the United States. The designation of "orphan drug" is applied because these medications target diseases that do not have enough prevalence to attract the necessary financial investments from pharmaceutical companies, hence the term "orphan," indicating that these diseases are often neglected by drug manufacturers. This designation allows for certain incentives, such as tax credits, market exclusivity, and grants, to encourage the development of treatments for these rare conditions. Consequently, the correct identification of an orphan drug as one designed for rare diseases clearly highlights its purpose and the regulatory framework that supports such medicines. The other options incorrectly describe the purpose or nature of an orphan drug. Common ailments would not fall into the category of rare diseases, hence do not qualify as orphan drugs. While a drug having no market competition or high annual sales may occur in certain cases, these factors alone do not pertain specifically to the definition of an orphan drug. The defining characteristic revolves solely around its target patient population and the rarity of the disease it addresses.

An orphan drug is defined as a medication specifically developed to treat a rare disease or condition, often affecting fewer than 200,000 individuals in the United States. The designation of "orphan drug" is applied because these medications target diseases that do not have enough prevalence to attract the necessary financial investments from pharmaceutical companies, hence the term "orphan," indicating that these diseases are often neglected by drug manufacturers.

This designation allows for certain incentives, such as tax credits, market exclusivity, and grants, to encourage the development of treatments for these rare conditions. Consequently, the correct identification of an orphan drug as one designed for rare diseases clearly highlights its purpose and the regulatory framework that supports such medicines.

The other options incorrectly describe the purpose or nature of an orphan drug. Common ailments would not fall into the category of rare diseases, hence do not qualify as orphan drugs. While a drug having no market competition or high annual sales may occur in certain cases, these factors alone do not pertain specifically to the definition of an orphan drug. The defining characteristic revolves solely around its target patient population and the rarity of the disease it addresses.