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Accelerating the Availability of Medications to Pediatric Patients by Optimizing the Use of Extrapolation of Efficacy

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Abstract

Improving pediatric therapeutic development is a mission of universal importance among health authorities, pharmaceutical companies, academic institutions, and healthcare professionals. Following the passage of legislation in the United States and Europe, we witnessed the most significant advancement yet in pediatric data generation, resulting in added pediatric use information to almost 700 product labels. Tools to accelerate generation of data for the pediatric population are available for use today, and when utilized in accordance with current practices and laws, these tools could increase the amount and timeliness of pediatric information available for clinicians and patients. If we utilize the current laws that allow regulators to incentivize and require evidence generation, apply extrapolation, and utilize modeling and simulation, as well as including adolescents in the pivotal studies alongside adults as appropriate, two strategic goals could be achieved by 2030: (1) reduce the time to pediatric approval by 50%, and (2) renew pediatric labeling information for 15 priority pediatric drugs without patent and/or exclusivity.

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Authors and Affiliations

  1. Center for Translational Medicine, University of Maryland School of Pharmacy, 20 North Pine Street, Baltimore, MD, 21201, USA

    Allison Dunn, Dawoon Jung & Jogarao V. S. Gobburu

  2. Merck, Rahway, NJ, USA

    Lisa L. Bollinger

  3. Janssen Research & Development, Spring House, PA, USA

    Michael Krams

  4. Early Development Analytics, Novartis Institutes of Biomedical Research, Cambridge, MA, USA

    Brian P. Smith

Authors
  1. Allison Dunn
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  2. Dawoon Jung
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  3. Lisa L. Bollinger
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  4. Michael Krams
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  5. Brian P. Smith
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  6. Jogarao V. S. Gobburu
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Contributions

All authors participated in the conception and viewpoint of the manuscript. DJ conducted the Vivpro RIA analysis. AD, JG, and DJ drafted the manuscript and LB, BS, and MK provided critical revisions. All authors contributed to the final version of the manuscript.

Corresponding author

Correspondence to Jogarao V. S. Gobburu.

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Conflict of interest

Dr. Jogarao V.S. Gobburu is a co-founder of Vivpro Corp and Pumas-AI.

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Dunn, A., Jung, D., Bollinger, L.L. et al. Accelerating the Availability of Medications to Pediatric Patients by Optimizing the Use of Extrapolation of Efficacy. Ther Innov Regul Sci 56, 873–882 (2022). https://doi.org/10.1007/s43441-022-00411-2

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  • DOI: https://doi.org/10.1007/s43441-022-00411-2

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