In what way do orphan drugs serve public health needs?

Orphan drugs are specifically developed to treat rare diseases, also known as orphan diseases, which typically affect a small percentage of the population. These diseases might otherwise be neglected by the pharmaceutical industry due to the limited market and low profitability associated with their treatment. By focusing on these rare conditions, orphan drugs fulfill a significant public health need by providing therapeutic options for patients who would otherwise have few or no treatment options available.

The designation of these drugs as “orphan” reflects the financial and research challenges in developing treatments for conditions that affect a smaller number of individuals. Thus, orphan drugs are pivotal in addressing the unmet medical needs of patients with rare diseases, ultimately improving their quality of life and providing hope for innovative treatments.