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Cardiovascular Paediatric Medicines Development: Have Paediatric Investigation Plans Lost Heart?

Bethany Faulkner1, M Begoña Delgado-Charro1

  • 1Department of Pharmacy and Pharmacology, University of Bath, Bath BA2 7AY, UK.

Pharmaceutics
|December 5, 2020
PubMed

Insights

European Paediatric Investigation Plans (PIPs) have not met most paediatric cardiovascular disease needs. Many PIPs resulted in waivers, leaving critical areas like heart failure untreated and increasing off-label drug use in children.

Area of Science:

  • Cardiovascular Medicine
  • Pediatric Pharmacology
  • Regulatory Science

Background:

  • The European Union Paediatric Regulation (2007) aimed to improve pediatric medicine development.
  • Paediatric cardiovascular diseases (CVD) represent a significant therapeutic area with specific needs.
  • Off-label use of cardiovascular medications in children remains a concern.

Purpose of the Study:

  • To evaluate the effectiveness of Paediatric Investigation Plans (PIPs) in addressing pediatric cardiovascular disease needs since 2007.
  • To identify unmet needs in pediatric cardiovascular medicine development.
  • To analyze trends in PIPs for cardiovascular drugs within the European Medicines Agency repository.

Main Methods:

  • Systematic search of the European Medicines Agency repository for PIPs.
  • Analysis of PIP decisions related to cardiovascular medicines.
  • Comparison of PIP outcomes with established pediatric cardiovascular needs.
  • Case study on valsartan to illustrate collaborative challenges.

Main Results:

  • Out of 1866 PIPs, 12% concerned cardiovascular medicines.
  • Only 14% of identified pediatric cardiovascular needs were met by PIPs with positive decisions.
  • 71.9% of cardiovascular PIP decisions were full waivers, exempting pediatric studies.
  • Significant unmet needs exist for pediatric heart failure and hypertension treatments.

Conclusions:

  • Despite overall PIP increases, pediatric cardiovascular needs remain largely unmet.
  • Waivers and lack of positive decisions contribute to continued off-label use of CVD drugs in children.
  • Collaboration between industry, regulators, and healthcare providers is crucial to reduce off-label prescribing and address pediatric CVD needs.

Keywords:
European Medicines Agencycardiovascularheart failurehypertensionoff-labelpaediatricpaediatric drug developmentpaediatric investigation planpharmaceuticalvalsartan

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