True about orphan drugs:
**Core Concept**
Orphan drugs are medications designed to treat rare diseases or conditions, typically affecting fewer than 200,000 people in the United States. The development and marketing of orphan drugs pose unique challenges due to the small market size and limited financial incentives.
**Why the Correct Answer is Right**
The Orphan Drug Act of 1983 provides incentives for pharmaceutical companies to develop orphan drugs, including tax credits, exemptions from certain FDA fees, and seven years of marketing exclusivity. This legislation aims to encourage the development of treatments for rare diseases, which are often neglected by the pharmaceutical industry due to their small market size. The Act also provides a streamlined regulatory process for orphan drug approval, allowing for faster access to these life-saving treatments.
**Why Each Wrong Option is Incorrect**
**Option A:** This option is incorrect because orphan drugs do not necessarily target diseases with a genetic basis. While many rare diseases have a genetic component, others may be caused by environmental or infectious factors.
**Option B:** This option is incorrect because orphan drugs are not necessarily more expensive than other medications. While the cost of developing and marketing orphan drugs may be higher due to the small market size, the prices of these medications can vary widely depending on the specific treatment and the company involved.
**Option C:** This option is incorrect because the Orphan Drug Act does not provide exclusive marketing rights for orphan drugs. While the Act does grant seven years of marketing exclusivity, this is not the same as exclusive marketing rights, which would prevent other companies from developing and marketing competing treatments.
**Clinical Pearl / High-Yield Fact**
To remember the key characteristics of orphan drugs, use the mnemonic **RARE**: Rare diseases, Alternative treatments, Regulatory incentives, and Economic challenges.
**Correct Answer:** D.