Progress in Drug Formulations for Rare and Orphan Diseases

Abstract

Rare and orphan diseases, often characterized by low prevalence and high unmet medical needs, present significant challenges for drug formulation and development. However, recent advancements in biotechnology, drug delivery systems, and regulatory support have made it possible to create more effective treatments for these conditions. This article reviews the progress made in drug formulations for rare and orphan diseases, focusing on the role of innovative drug delivery systems, gene therapies, and biopharmaceuticals in overcoming these challenges. The article also examines current trends in regulatory frameworks that facilitate the development of orphan drugs.