Pediatric studies and labeling additions required by the U.S. FDA for novel drugs approved from 2011 to 2023: A retrospective cohort study
PREA was implemented to advance pediatric drug research and fill a critical gap in pediatric labeling of new drugs. However, our findings reveal frequent delays in study completion and labeling updates, with just over half of labeling additions completed 10 years after drug approval. Strengthening reporting requirements and expanding the FDA's enforcement authority are essential to ensuring that children receive timely access to safe and effective therapies supported by high-quality evidence.
Authors
- Anita Katharina Wagner (ORCID: https://orcid.org/0000-0002-1069-6668)
- Huseyin Naci (ORCID: https://orcid.org/0000-0002-7192-5751)
- Maximilian Siebert (ORCID: https://orcid.org/0000-0003-4385-5773)
- Alba Gutiérrez‐Sacristán (ORCID: https://orcid.org/0000-0002-1245-198X)
- Florence T. Bourgeois (ORCID: https://orcid.org/0000-0001-7798-4560)
- Michelle Ouviña (ORCID: https://orcid.org/0009-0009-6567-3162)
- Rylee McGonigle
Institutions
- Boston Children's Hospital (US)
- Harvard University (US)
- Harvard Pilgrim Health Care (US)
- Center for Systems Biology (US)
- London School of Economics and Political Science (GB)
Publication Details
- Journal
- PLoS Medicine
- Published
- 2025-12-17
- DOI
- https://doi.org/10.1371/journal.pmed.1004651
- Citations
- 1
- Primary Topic
- Pharmaceutical studies and practices
- Type
- article
- Field-Weighted Citation Impact
- 3.49
Funders
- Arnold Ventures