What term describes medications designated for rare diseases or conditions?

The term that describes medications designated for rare diseases or conditions is orphan drugs. These are specifically developed to treat conditions that affect a small percentage of the population, and they may not be financially viable for manufacturers to produce without incentives. The term "orphan" refers to the fact that these drugs are developed for "orphaned" conditions that often lack sufficient market potential for pharmaceutical companies, leading to initiatives that encourage their development, such as federal grants or tax incentives.

Orphan drugs are significant in the context of the healthcare system because they address unmet medical needs for patients with rare diseases. Understanding this term is essential for professionals in the healthcare and pharmaceutical fields, especially those involved in drug distribution and access programs like the 340B Program, which aims to enhance patient access to necessary medications, including orphan drugs.

In contrast, the other terms listed do not specifically refer to medications for rare diseases. 340B drugs pertain to the pricing and distribution mechanisms in the 340B Drug Pricing Program, essential medications refer to drugs that are considered vital for maintaining health and treating common conditions, and specialty drugs typically refer to high-cost medications that are used to treat complex or chronic diseases, which may not necessarily be for rare conditions.