Orphan Drug Applications: Navigating the Path to Approval provides a comprehensive guide for understanding the intricacies of orphan drug development, from the initial designation process to final market approval. Designed for both professionals and students in the pharmaceutical and biotech industries, this book demystifies the regulatory landscape surrounding orphan drugs and highlights the incentives available to encourage innovation for rare diseases. Through a clear exploration of the Orphan Drug Act, global regulations, and key challenges such as clinical trials and patient access, this book equips readers with the knowledge needed to navigate the complexities of developing life-saving treatments for underserved patient populations.