Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs

While artificial intelligence has successful and innovative applications in common medicine, could its application facilitate research on rare diseases? This study explores the application of artificial intelligence (AI) in orphan drug research, focusing on how AI can address three major barriers: high financial risk, development complexity, and low trialability. This paper begins with an overview of orphan drug development and AI applications, defining key concepts and providing a background on the regulatory framework of and AI’s role in medical research. Next, it examines how AI can lower financial risks by streamlining drug discovery and development processes, analyzing complex data, and predicting outcomes to improve our understanding of rare diseases. This study then explores how AI can enhance clinical trials through simulations and virtual trials, compensating for the limited patient populations available for rare disease research. Finally, it discusses the broader implications of integrating AI in orphan drug development, emphasizing the potential for AI to accelerate drug discovery and improve treatment success rates, and highlights the need for ongoing innovation and regulatory support to maximize the benefits of AI-driven research in healthcare. Based on those results, we discuss the implications for traditional and AI-powered business in the drug industry.