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Impact of Regulatory Incentive Programs on the Future of Pediatric Drug Development

  • Special Section: Pediatric Therapeutic Development: Review Article
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An Erratum to this article was published on 01 November 2019

This article has been updated

Abstract

Surveys evaluating industry experience with performing pediatric studies under the Best Pharmaceutical for Children Act (BPCA) and Pediatric Research Equity Act (PREA) regulatory regime were conducted by Tufts Center for the Study of Drug Development (Tufts CSDD) in 2000, 2006, and 2016. These survey results are being used to assess the future impact of regulatory incentive programs on generating pediatric specific labeling information and development of age-appropriate drug formulations. A second perspective will be provided through the experience and expertise of neonatal/pediatric clinicians and researchers with a focus on the urgent need for the study of new and existing drugs in this vulnerable population (especially with 90% of drugs in neonates still being used off-label). This group will also address the impact of existing regulations and the likely trajectory of future pediatric drug development efforts after nearly 2 decades of regulatory incentives (both mandatory and voluntary). Finally, this review will provide input on approaches that are needed to continue to advance pediatric drug development with an emphasis on rare diseases.

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References

  1. Milne C-P. Exploring the frontiers of law and science: FDAMA’s pediatric studies incentive. Food Drug Law J. 2002;57:491–517.

    PubMed  Google Scholar 

  2. Kaitin KI, ed. Pediatric Study Costs Increased 8-Fold Since 2000 as Complexity Level Grew. Boston, MA: Tufts Impact Report; March/April 2007. Louis Lasagna Library, Tufts CSDD, Boston, MA; rachel.stanton@tufts.edu.

  3. Kaitin KI, ed. Pediatric Regulatory Science, Clinical Trial Networks Hold Promise for Studies. Boston, MA: Tufts Impact Report; November/December 2016. Louis Lasagna Library, Tufts CSDD, Boston, MA; rachel.stanton@tufts.edu.

  4. Wang J, Avant D, Green D, et al. A survey of neonatal pharmacokinetic and pharmacodynamics studies in pediatric drug development. Clin Pharmacol Ther. 2015;98:328–335.

    Article  CAS  Google Scholar 

  5. Laughon MM, Avant D, Tripathi N, et al. Drug labeling and exposure in neonates. JAMA Pediatr. 2014;168:130–136.

    Article  Google Scholar 

  6. Stiers JL, Ward R. Newborns, one of the last therapeutic orphans to be adopted. JAMA Pediatr. 2014;168:106–108.

    Article  Google Scholar 

  7. Buckley LA, Salunke S, Thompson K, Baer G, Fegley D, Turner MA. Challenges and strategies to facilitate formulation development of pediatric drug products: Safety qualification of excipients. Int J Pharm. 2018; 536:563–569.

    Article  CAS  Google Scholar 

  8. The impact of premature birth on society. March of dimes. https://www.marchofdimes.org/mission/the-economic-and-societal-costs.aspx. Accessed August 5, 2018.

  9. Simeoni U, Armengaud JB, Siddeek B, Tolsa JF. Perinatal origins of adult disease. Neonatology. 2018;113:393–399.

    Article  Google Scholar 

  10. Davis JM, Turner MA. Global collaboration to develop new and existing drugs for neonates. JAMA Pediatr. 2015;169:887–888.

    Article  Google Scholar 

  11. Turner MA, Portman RJ, Davis JM. Regulatory science in neonates: a framework that supports evidence-based drug therapy. JAMA Pediatr. 2017;171:721–722.

    Article  Google Scholar 

  12. Offringa M, Davis JM, Turner MA, et al. Applying regulatory science to develop safe and effective medicines for neonates: report of the US Food and Drug Administration first annual neonatal scientific workshop, October 28–29, 2014. Therapeutic Innovation & Regulatory Science. 2015;49:623–631.

    Article  Google Scholar 

  13. Denne SC, Davis JM. Improving pharmaceutical trials for children: a call to the pediatric academic community. Pediatr Res. 2018;84:319–320.

    Article  Google Scholar 

  14. Smith AM, Davis JM. Challenges and opportunities to enhance global drug development in neonates. Curr Opin Pediatr. 2017; 29:149–152.

    Article  Google Scholar 

  15. Stoll BJ, Hansen NI, Bell EF, et al. Trends in care practices, morbidity, and mortality of extremely preterm neonates, 1993–2012. JAMA. 2015;314:1039–1051.

    Article  CAS  Google Scholar 

  16. Steinhorn R, Davis JM, Göpel W, et al; for the International Neonatal Consortium. Chronic pulmonary insufficiency of prematurity: developing optimal endpoints for drug development. J Pediatr. 2017;191:15–21.

    Article  Google Scholar 

  17. Murray PD, Bier BE, Hirschfeld S, Klein AK, Davis JM. Assessment of a shortened informed consent form for pediatric research: a pilot study. Pediatr Res. 2018;84(4):516–519.

    Article  Google Scholar 

  18. Davis JM, Baer GR, Portman R, et al. Enrollment of neonates in more than one clinical trial. Clin Ther. 2017;39:1959–1969.

    Article  Google Scholar 

  19. Allegaert K, van den Anker J. Neonatal drug therapy: the first frontier of therapeutics in children. Clin Pharmacol Ther. 2015; 98:288–297.

    Article  CAS  Google Scholar 

  20. Institute for Advanced Clinical Trials for Children. https://www.iactc.org/. Accessed July 22, 2018.

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Authors and Affiliations

  1. Department of Pediatrics, The Floating Hospital for Children at Tufts Medical Center, Boston, MA, USA

    Elizabeth Yen MD & Jonathan M. Davis MD

  2. The Tufts Clinical and Translational Research Institute, Boston, MA, USA

    Jonathan M. Davis MD

  3. The Tufts Center for the Study of Drug Development, Boston, MA, USA

    Christopher-Paul Milne DVM, MPH, JD

  4. Division of Newborn Medicine, The Floating Hospital for Children at Tufts Medical Center, 800 Washington Street, Boston, MA, 02111, USA

    Elizabeth Yen MD

Authors
  1. Elizabeth Yen MD
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  2. Jonathan M. Davis MD
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  3. Christopher-Paul Milne DVM, MPH, JD
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Correspondence to Elizabeth Yen MD.

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Yen, E., Davis, J.M. & Milne, CP. Impact of Regulatory Incentive Programs on the Future of Pediatric Drug Development. Ther Innov Regul Sci 53, 609–614 (2019). https://doi.org/10.1177/2168479019837522

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  • DOI: https://doi.org/10.1177/2168479019837522

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