What distinguishes orphan drugs from other types of medications?

Orphan drugs are specifically designed to treat rare diseases or conditions, which is what makes them distinct from other types of medications. The term "orphan" refers to the fact that these drugs are often developed to address medical needs that are not typically met due to the limited patient population, making them less financially attractive for pharmaceutical companies to produce without regulatory incentives. This designation ensures that medications are available for conditions that might otherwise be overlooked in favor of treatments for more common diseases.

The other options do not accurately reflect the nature of orphan drugs. For example, while some medications are used in hospitals, not all orphan drugs are restricted to this environment. Additionally, orphan drugs are not confined to 340B pricing; they can be available through various pricing structures. Lastly, orphan drugs are not necessarily generic versions of existing medications; they are distinct compounds developed specifically to treat rare conditions. This unique focus on rare diseases is what fundamentally sets orphan drugs apart in the pharmaceutical landscape.