The Pediatric Drug Discovery Pipeline
Main Article Content
The pediatric drug discovery pipeline is a vital part of pharmaceutical research, focusing on developing and optimizing medications specifically for children. This process differs significantly from adult drug development due to age-related physiological differences, developmental changes, and the need for age-appropriate formulations. Key stages include preclinical studies, which assess drug mechanisms and safety in non-human models, and clinical trials that evaluate efficacy and safety in pediatric patients. Despite its importance, pediatric drug development faces substantial challenges, including limited clinical trial data, ethical concerns in testing on minors, and strict regulatory requirements for pediatric-specific studies. Studies indicate that only 50–60% of drugs used in pediatrics have been formally tested in children, underscoring a critical gap in evidence-based pharmacotherapy. The process is further complicated by the need for precise dosing across various age groups, requiring specialized formulations. Recent advancements aim to address these challenges. Over the past decade, pediatric clinical trials have increased by 20%, driven by regulatory incentives, technological innovations in drug delivery, and stronger collaborations between research institutions, industry, and regulators. The adoption of pediatric-specific trial designs and improved data-sharing mechanisms has further enhanced development efforts. From a policy and industry perspective, strengthening incentives, streamlining regulatory pathways, and fostering interdisciplinary research are essential to accelerating pediatric drug innovation. This review highlights the evolving landscape of pediatric drug development, emphasizing the need for continuous improvement to ensure that children receive safe, effective, and appropriately dosed medications.
Bradford, A. (2020). The Brussels effect: How the European Union rules the world. Oxford University Press.
Burrows, J. N., Duparc, S., Gutteridge, W. E., Hooft van Huijsduijnen, R., Kaszubska, W., Macintyre, F., Mazzuri, S., Möhrle, J. J., & Wells, T. N. C. (2017). New developments in anti-malarial target candidate and product profiles. Malaria Journal, 16, 1–29.
Chouhan, D., Dey, N., Bhardwaj, N., & Mandal, B. B. (2019). Emerging and innovative approaches for wound healing and skin regeneration: Current status and advances. Biomaterials, 216, 119267. https://doi.org/10.1016/j.biomaterials.2019.119267
Deppisch, L. M. (2025). The White House Physician: A History from George Washington to Joe Biden. McFarland.
Drakeman, D. L., Drakeman, L. N., & Oraiopoulos, N. (2022). From breakthrough to blockbuster: the business of biotechnology. Oxford University Press.
Elbadawi, M., McCoubrey, L. E., Gavins, F. K. H., Ong, J. J., Goyanes, A., Gaisford, S., & Basit, A. W. (2021). Harnessing artificial intelligence for the next generation of 3D printed medicines. Advanced Drug Delivery Reviews, 175, 113805. https://doi.org/10.1016/j.addr.2021.05.015
Hulleck, A. A., Menoth Mohan, D., Abdallah, N., El Rich, M., & Khalaf, K. (2022). Present and future of gait assessment in clinical practice: Towards the application of novel trends and technologies. Frontiers in Medical Technology, 4, 901331.
Jespersen, L., & Wallace, C. A. (2017). Triangulation and the importance of establishing valid methods for food safety culture evaluation. Food Research International, 100, 244–253. https://doi.org/10.1016/j.foodres.2017.07.009
Kapoor, D. U., Garg, R., Gaur, M., Patel, M. B., Minglani, V. V., Prajapati, B. G., Huanbutta, K., Sangnim, T., & Sriamornsak, P. (2024). Pediatric drug delivery challenges: Enhancing compliance through age-appropriate formulations and safety measures. Journal of Drug Delivery Science and Technology, 96, 105720. https://doi.org/10.1016/j.jddst.2024.105720
Kingma, E., & Finn, S. (2020). Neonatal incubator or artificial womb? distinguishing ectogestation and ectogenesis using the metaphysics of pregnancy. Bioethics, 34(4), 354–363.
Kwon, B. C., Choi, M.-J., Kim, J. T., Choi, E., Kim, Y. Bin, Kwon, S., Sun, J., & Choo, J. (2018). Retainvis: Visual analytics with interpretable and interactive recurrent neural networks on electronic medical records. IEEE Transactions on Visualization and Computer Graphics, 25(1), 299–309.
Lietzan, E. (2018). Access before Evidence and the Price of the FDA’s New Drug Authorities. U. Rich. L. Rev., 53, 1243.
Lloyd, J., & Cheyne, J. (2017). The origins of the vaccine cold chain and a glimpse of the future. Vaccine, 35(17), 2115–2120. https://doi.org/10.1016/j.vaccine.2016.11.097
McCuistion, L. E., DiMaggio, K. V., Winton, M. B., & Yeager, J. J. (2021). Pharmacology E-Book: A Patient-Centered Nursing Process Approach. Elsevier Health Sciences.
Mittal, P., Chopra, H., Kaur, K. P., & Gautam, R. K. (2023). New drug discovery pipeline. In Computational Approaches in Drug Discovery, Development and Systems Pharmacology (pp. 197–222). Elsevier. https://doi.org/10.1016/B978-0-323-99137-7.00003-4
Mohs, R. C., & Greig, N. H. (2017). Drug discovery and development: Role of basic biological research. Alzheimer’s & Dementia: Translational Research & Clinical Interventions, 3(4), 651–657. https://doi.org/10.1016/j.trci.2017.10.005
O’Daniel, J. M., Ackerman, S., Desrosiers, L. R., Rego, S., Knight, S. J., Mollison, L., Byfield, G., Anderson, K. P., Danila, M. I., Horowitz, C. R., Joseph, G., Lamoure, G., Lindberg, N. M., McMullen, C. K., Mittendorf, K. F., Ramos, M. A., Robinson, M., Sillari, C., & Madden, E. B. (2022). Integration of stakeholder engagement from development to dissemination in genomic medicine research: Approaches and outcomes from the CSER Consortium. Genetics in Medicine, 24(5), 1108–1119. https://doi.org/10.1016/j.gim.2022.01.008
Pagliusi, S., Ting, C.-C., Lobos, F., Datla, M., Gao, S., Homma, A., Meng, L., Suhardono, M., Suri, R., & Tippoo, P. (2017). Vaccines: Shaping global health. Vaccine, 35(12), 1579–1585. https://doi.org/10.1016/j.vaccine.2017.02.017
Reed, A. (2018). Without justice for all: The new liberalism and our retreat from racial equality. Routledge.
Synott, J. P. (2017). Teacher unions, social movements and the politics of education in Asia: South Korea, Taiwan and the Philippines. Routledge.
