Arizona MPJE (Pharmacy Jurisprudence) Practice Exam

Drugs used to treat rare diseases must affect fewer than how many patients to qualify under the Orphan Drug Act?

1. 100,000 patients

2. 150,000 patients

3. 200,000 patients

4. 250,000 patients

The correct answer is: 100,000 patients

The Orphan Drug Act was established to encourage the development of drugs for rare diseases. Under this legislation, a condition is classified as "orphan" if it affects less than 200,000 individuals in the United States. This threshold means that a drug targeting such a population primarily falls under the orphan designation. Therefore, the choice indicating fewer than 100,000 patients is relevant in the context of the act because it represents a significantly smaller group within the broader categorization of rare diseases, emphasizing the need for targeted treatments for conditions affecting an even smaller population. Overall, the Orphan Drug Act provides incentives for pharmaceutical companies to develop and market medications for these rare disorders, which might otherwise be overlooked due to limited market potential.