Clinical Trials · Pharmacology

What is the purpose of an 'Orphan Drug' designation?

  1. To expedite the approval of drugs used for pediatric patients
  2. To encourage the development of drugs for rare diseases affecting a small portion of the population
  3. To provide drugs for free to patients who cannot afford them
  4. To label drugs that have lost their patent protection
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Correct answer: To encourage the development of drugs for rare diseases affecting a small portion of the population

The Orphan Drug Act provides incentives, such as tax credits and extended market exclusivity, for pharmaceutical companies to develop treatments for rare diseases. These diseases are defined in the US as affecting fewer than 200,000 people.

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