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: h...

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Main Authors: Carla Irissarry, Thierry Burger-Helmchen
Format: Article
Language:English
Published: MDPI AG 2024-09-01
Series:Businesses
Subjects:
Online Access:https://www.mdpi.com/2673-7116/4/3/28
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author Carla Irissarry
Thierry Burger-Helmchen
author_facet Carla Irissarry
Thierry Burger-Helmchen
author_sort Carla Irissarry
collection DOAJ
description 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.
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spelling doaj-art-e3739444cd2d45b3bbb49c7e5d4761422025-08-20T01:56:13ZengMDPI AGBusinesses2673-71162024-09-014345347210.3390/businesses4030028Using Artificial Intelligence to Advance the Research and Development of Orphan DrugsCarla Irissarry0Thierry Burger-Helmchen1Paris School of Economics, 75014 Paris, FranceUniversité de Strasbourg, Université de Lorraine, AgroParisTech, CNRS, INRAE, BETA, 67000 Strasbourg, FranceWhile 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.https://www.mdpi.com/2673-7116/4/3/28AIresearchorphan drugrare diseasedrug discovery and industrybusiness
spellingShingle Carla Irissarry
Thierry Burger-Helmchen
Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs
Businesses
AI
research
orphan drug
rare disease
drug discovery and industry
business
title Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs
title_full Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs
title_fullStr Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs
title_full_unstemmed Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs
title_short Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs
title_sort using artificial intelligence to advance the research and development of orphan drugs
topic AI
research
orphan drug
rare disease
drug discovery and industry
business
url https://www.mdpi.com/2673-7116/4/3/28
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