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

Elizabeth Yen1, Jonathan M Davis1,2, Christopher-Paul Milne3

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

Therapeutic Innovation & Regulatory Science
|April 16, 2019
PubMed

Insights

Industry surveys reveal the impact of the Best Pharmaceutical for Children Act (BPCA) and Pediatric Research Equity Act (PREA) on pediatric drug development. This analysis informs future strategies for pediatric labeling and formulation, especially for rare diseases.

Area of Science:

  • Pharmacology and Therapeutics
  • Regulatory Science
  • Pediatric Medicine

Background:

  • Industry surveys conducted in 2000, 2006, and 2016 by Tufts CSDD assessed pediatric study experiences under BPCA and PREA.
  • A significant gap remains, with approximately 90% of drugs used off-label in neonates, highlighting the need for more research.

Purpose of the Study:

  • To evaluate the impact of regulatory incentives on pediatric drug labeling and formulation development.
  • To gather insights from clinicians and researchers on the trajectory of pediatric drug development.
  • To identify approaches for advancing pediatric drug development, particularly for rare diseases.

Main Methods:

  • Analysis of survey data from the Tufts Center for the Study of Drug Development (Tufts CSDD) from 2000, 2006, and 2016.
  • Incorporation of expert perspectives from neonatal/pediatric clinicians and researchers.
  • Review of existing regulations and their influence on pediatric drug development.

Main Results:

  • Survey results are informing assessments of future regulatory impacts on pediatric labeling and age-appropriate drug formulations.
  • Expert insights highlight the urgent need for studies on new and existing drugs in vulnerable pediatric populations.
  • Nearly two decades of regulatory incentives (mandatory and voluntary) have shaped pediatric drug development.

Conclusions:

  • Continued advancement in pediatric drug development requires strategic input and focus on rare diseases.
  • Addressing off-label drug use in neonates is critical.
  • Future pediatric drug development will be influenced by regulatory frameworks and clinical expertise.

Keywords:
clinical trialsdrug developmentpediatric and neonatal studiesrare diseasesregulatory incentives

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