Question

What do you mean by Orphan Drug?

• A. A drug for a disease which is not having any other treatment options at all
• B. A drug which is useful for rare disease
• C. A drug that is available in abundance
• D. A drug meant to be distributed among the orphans who can not afford the cost of the drug

Answer 1

The Correct Option is B

An orphan drug is defined as a pharmaceutical agent that has been developed specifically to treat a rare medical condition, often referred to as an "orphan disease." These diseases are termed "orphan" because they affect a small percentage of the population, making it economically unviable for pharmaceutical companies to develop medications without assistance or incentives from the government.

Now, let's evaluate each option:

1. A drug for a disease which is not having any other treatment options at all: This statement can be misleading as orphan drugs are not defined by the availability of other treatment options, but rather by the rarity of the diseases they treat.
2. A drug which is useful for rare disease: This is correct because orphan drugs are specifically aimed at treating rare conditions, which typically affect a smaller segment of the population.
3. A drug that is available in abundance: Orphan drugs, by definition, are not widespread and are targeted for specific rare conditions, making this option incorrect.
4. A drug meant to be distributed among the orphans who cannot afford the cost of the drug: While humanitarian in concept, this option does not accurately describe an orphan drug. It does not address the aspect of treating rare diseases.

In conclusion, the correct answer is A drug which is useful for rare disease.

This understanding aligns with the Orphan Drug Act of 1983 in the USA, which was established to encourage the development of drugs for rare diseases by providing benefits such as tax credits and marketing exclusivity.