IDEAS home Printed from https://ideas.repec.org/a/gam/jbusin/v4y2024i3p28-472d1474478.html
   My bibliography  Save this article

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

Author

Listed:
  • Carla Irissarry

    (Paris School of Economics, 75014 Paris, France)

  • Thierry Burger-Helmchen

    (Université de Strasbourg, Université de Lorraine, AgroParisTech, CNRS, INRAE, BETA, 67000 Strasbourg, France)

Abstract

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.

Suggested Citation

  • Carla Irissarry & Thierry Burger-Helmchen, 2024. "Using Artificial Intelligence to Advance the Research and Development of Orphan Drugs," Businesses, MDPI, vol. 4(3), pages 1-20, September.
    • Handle: RePEc:gam:jbusin:v:4:y:2024:i:3:p:28-472:d:1474478
    as

    Download full text from publisher

    File URL: https://www.mdpi.com/2673-7116/4/3/28/pdf
    Download Restriction: no
    File URL: https://www.mdpi.com/2673-7116/4/3/28/
    Download Restriction: no
    ---><---

    References listed on IDEAS

    as
    1. Wu, Qiang & He, Qile, 2020. "DIY Laboratories and business innovation ecosystems: The case of pharmaceutical industry," Technological Forecasting and Social Change, Elsevier, vol. 161(C).
    2. Garza Ramos, Alejandro & Daim, Tugrul & Gaats, Lukas & Hutmacher, Dietmar W. & Hackenberger, David, 2022. "Technology roadmap for the development of a 3D cell culture workstation for a biomedical industry startup," Technological Forecasting and Social Change, Elsevier, vol. 174(C).
    3. Burger-Helmchen, Thierry, 2009. "Option chain and change management: A structural equation application," European Management Journal, Elsevier, vol. 27(3), pages 176-186, June.
    4. Rita Gunther McGrath & Atul Nerkar, 2004. "Real options reasoning and a new look at the R&D investment strategies of pharmaceutical firms," Strategic Management Journal, Wiley Blackwell, vol. 25(1), pages 1-21, January.
    Full references (including those not matched with items on IDEAS)

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Nault, Kelly A. & Sezer, Ovul & Klein, Nadav, 2023. "It’s the journey, not just the destination: Conveying interpersonal warmth in written introductions," Organizational Behavior and Human Decision Processes, Elsevier, vol. 177(C).
    2. Estrada, Isabel & de la Fuente, Gabriel & Martín-Cruz, Natalia, 2010. "Technological joint venture formation under the real options approach," Research Policy, Elsevier, vol. 39(9), pages 1185-1197, November.
    3. Raisa Pérez-Vas & Félix Puime Guillén & Joaquín Enríquez-Díaz, 2021. "Valuation of a Company Producing and Trading Seaweed for Human Consumption: Classical Methods vs. Real Options," IJERPH, MDPI, vol. 18(10), pages 1-13, May.
    4. Bouncken, Ricarda B. & Qiu, Yixin & García, F. Javier Sendra, 2021. "Flexible pattern matching approach: Suggestions for augmenting theory evolvement," Technological Forecasting and Social Change, Elsevier, vol. 167(C).
    5. Jan Hendrik, Fisch, 2011. "Real call options to enlarge foreign subsidiaries - The moderating effect of irreversibility on the influence of economic volatility and political instability on subsequent FDI," Journal of World Business, Elsevier, vol. 46(4), pages 517-526, October.
    6. Jeffrey J. Reuer & Tony W. Tong, 2007. "Corporate investments and growth options," Managerial and Decision Economics, John Wiley & Sons, Ltd., vol. 28(8), pages 863-877.
    7. Van De Vrande, Vareska & Subramanian, Annapoornima M. & Lévesque, Moren & Klopf, Patricia, 2024. "The interdependent influence of lobbying and intellectual capital on new drug development," Research Policy, Elsevier, vol. 53(2).
    8. Lu, Chao & Yang, Meng & Xia, Xiaoxue, 2023. "Economic policy uncertainty and default risk: Evidence from China," Economic Analysis and Policy, Elsevier, vol. 79(C), pages 821-836.
    9. Zhang, Min & Liu, Yaosong & Xie, Lu & Ye, Tingting, 2017. "Does the cutoff of “red capital” raise a red flag? Political connections and stock price crash risk," The North American Journal of Economics and Finance, Elsevier, vol. 39(C), pages 89-109.
    10. Carol Alexander & Xi Chen, 2021. "Model risk in real option valuation," Annals of Operations Research, Springer, vol. 299(1), pages 1025-1056, April.
    11. Taj, Saud A., 2016. "Application of signaling theory in management research: Addressing major gaps in theory," European Management Journal, Elsevier, vol. 34(4), pages 338-348.
    12. Hartmann, Marcus & Hassan, Ali, 2006. "Application of real options analysis for pharmaceutical R&D project valuation--Empirical results from a survey," Research Policy, Elsevier, vol. 35(3), pages 343-354, April.
    13. Wang, Tao, 2023. "Toward an understanding of innovation failure: The timing of failure experience," Technovation, Elsevier, vol. 125(C).
    14. Simon J. D. Schillebeeckx & Sankalp Chaturvedi & Gerard George & Zella King, 2016. "What do i want? The effects of individual aspiration and relational capability on collaboration preferences," Strategic Management Journal, Wiley Blackwell, vol. 37(7), pages 1493-1506, July.
    15. Fatima Shuwaikh & Mathew Hughes & Souad Brinette & Sabrina Khemiri, 2024. "Investment decisions under uncertainty: Corporate venture capital as a real option," International Journal of Finance & Economics, John Wiley & Sons, Ltd., vol. 29(2), pages 2452-2471, April.
    16. Marc Fréchet & Hassen Raîs, 2015. "Les managers raisonnent-ils par options réelles ? Une étude exploratoire des déterminants," Post-Print hal-01764120, HAL.
    17. Lu, Hsi-Peng & Cheng, Hsiang-Ling & Tzou, Jen-Chuen & Chen, Chiao-Shan, 2023. "Technology roadmap of AI applications in the retail industry," Technological Forecasting and Social Change, Elsevier, vol. 195(C).
    18. Rammer, Christian, 2012. "Schwerpunktbericht zur Innovationserhebung 2010: Management von Innovationsprojekten - Auswirkungen der Wirtschaftskrise," ZEW Dokumentationen 12-03, ZEW - Leibniz Centre for European Economic Research.
    19. Sam Ransbotham & Sabyasachi Mitra, 2010. "Target Age and the Acquisition of Innovation in High-Technology Industries," Management Science, INFORMS, vol. 56(11), pages 2076-2093, November.
    20. Yang, Hongyan & Steensma, H. Kevin, 2014. "When do firms rely on their knowledge spillover recipients for guidance in exploring unfamiliar knowledge?," Research Policy, Elsevier, vol. 43(9), pages 1496-1507.

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:gam:jbusin:v:4:y:2024:i:3:p:28-472:d:1474478. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: MDPI Indexing Manager (email available below). General contact details of provider: https://www.mdpi.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.