FPGEE 2025 Practice Exam for NABP – The Complete All-in-One Guide to Exam Success!

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What is the primary purpose of orphan drugs?

A special category of drugs designed for widespread use

A special category of drugs to treat patients with rare diseases

The primary purpose of orphan drugs is to treat patients with rare diseases. These drugs are developed specifically to address conditions that affect a small percentage of the population, often referred to as orphan diseases. The market for these conditions is typically not attractive for pharmaceutical companies due to the limited number of patients, which is why orphan drugs receive special designation and incentives to encourage their development.

This designation helps ensure that individuals with rare diseases have access to necessary treatments, which might not otherwise be commercially viable. The development and approval of orphan drugs are supported by regulatory pathways that offer benefits like tax credits, grants, and extended market exclusivity, recognizing the need for therapies in underserved patient populations.

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Prescription drugs recognized by the FDA

Drugs that are available without a prescription

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