Challenges in Orphan Drug Development: Addressing Regulatory, Economic, and Scientific Hurdles for Rare Disease Treatments

Abstract

Orphan drugs are essential for treating rare diseases, but their development faces unique regulatory, economic, and scientific challenges. This paper examines these challenges using a survey of 150 respondents—comprised of pharmaceutical industry professionals, regulatory experts, and rare disease researchers. The data, analyzed via T-tests, ANOVA, correlation, and regression, reveals significant hurdles in orphan drug development. The analysis shows differences in perceptions between stakeholders and explores the impact of collaboration on the speed of drug approvals. Policy reforms and increased incentives are suggested to overcome these challenges.