According to the FDA, what does the term "orphan drug" refer to?

According to the FDA, what does the term "orphan drug" refer to?

• A. A drug that is only for pediatric use
• B. A drug developed for a rare disease affecting fewer than 200,000 people
• C. A drug that is not approved for any indications
• D. A drug that is readily available over the counter

Answer: (B)

The term "orphan drug" specifically refers to a drug that is developed to treat a rare disease or condition affecting fewer than 200,000 individuals in the United States. This classification is critical because developing treatments for rare diseases often presents challenges, such as limited financial incentives for pharmaceutical companies. To encourage the development of these drugs, the FDA provides certain incentives, including tax credits and market exclusivity for a set period of time once the drug is approved. Understanding this term within the framework of FDA regulations highlights the importance of addressing unmet medical needs, as many rare diseases may otherwise go without effective treatments due to their limited patient bases. Additionally, orphan drugs often receive special designations and support from regulatory agencies to facilitate their development and approval.

The term "orphan drug" specifically refers to a drug that is developed to treat a rare disease or condition affecting fewer than 200,000 individuals in the United States. This classification is critical because developing treatments for rare diseases often presents challenges, such as limited financial incentives for pharmaceutical companies. To encourage the development of these drugs, the FDA provides certain incentives, including tax credits and market exclusivity for a set period of time once the drug is approved.

Understanding this term within the framework of FDA regulations highlights the importance of addressing unmet medical needs, as many rare diseases may otherwise go without effective treatments due to their limited patient bases. Additionally, orphan drugs often receive special designations and support from regulatory agencies to facilitate their development and approval.