Assessing Information Gaps Associated with Initial Pediatric Study Plans for New Oncology Drug and Biological Products.
Journal
Clinical pharmacology and therapeutics
ISSN: 1532-6535
Titre abrégé: Clin Pharmacol Ther
Pays: United States
ID NLM: 0372741
Informations de publication
Date de publication:
09 2023
09 2023
Historique:
received:
27
02
2023
accepted:
19
06
2023
medline:
21
8
2023
pubmed:
9
7
2023
entrez:
9
7
2023
Statut:
ppublish
Résumé
The Research Acceleration for Cure and Equity (RACE) for Children Act requires sponsors to submit a Pediatric Study Plan (PSP) with a proposed pediatric investigation of new molecularly targeted drugs and biologics that are intended for treatment of adult cancers, and whose target is relevant to pediatric cancer or provide a justification for a plan to request a deferral or waiver of the required investigation. A landscape analysis was performed to identify trends in information gaps associated with a sponsor's first initial PSP (iPSP) submission for oncologic new molecular entities received in 2021. Comments sent to sponsors by the US Food and Drug Administration (FDA) during the review process of each evaluated iPSP were categorized using nine flags relating to different portions of the PSP. For iPSPs that included a plan for a full waiver request, the most common information gap was inadequate justification based on molecular target relevance. All other sponsor proposed plans (deferral and/or partial waiver or investigation) were found to have information gaps related to clinical study features, clinical pharmacology, and/or missing clinical or nonclinical data. This landscape analysis of iPSPs shows the trends in comments that often occur during initial review and may help to provide sponsors with more direction for preparing an adequate iPSP to fulfill statutory requirements aimed at ensuring pediatric patients are considered in the development of new molecularly targeted drugs.
Substances chimiques
Biological Products
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
618-622Informations de copyright
© 2023 The Authors. Clinical Pharmacology & Therapeutics © 2023 American Society for Clinical Pharmacology and Therapeutics.
Références
Reaman, G. et al. Accelerating the global development of pediatric cancer drugs: a call to coordinate the submissions of pediatric investigation plans and pediatric study plans to the European Medicines Agency and US Food and Drug Administration. J. Clin. Oncol. 38, 4227-4230 (2020).
US Food and Drug Administration FDA Reauthorization Act of 2017 (FDARA). <https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/fdareauthorization-act-2017-fdara>.
505B(e)(2)(A) of the FD&C Act 21 U.S.C. 355c(e)(2)(A). <https://uscode.house.gov/view.xhtml?req=granuleid:USC-prelim-title21-section355c&num=0&edition=prelim>.
U.S. Food and Drug Administration. Pediatric Study Plans: Content of and Process for Submitting Initial Pediatric Study Plans and Amended Initial Pediatric Study Plans Guidance for Industry <https://www.fda.gov/media/86340/download> (2020).
U.S. Food and Drug Administration. FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act <https://www.fda.gov/media/133440/download> (2020).
U.S. Food and Drug Administration. Pediatric Oncology <https://www.fda.gov/about-fda/oncology-center-excellence/pediatric-oncology>.
Charlab, R., Burckart, G.J. & Reaman, G.H. Fine-tuning the relevance of molecular targets to pediatric cancer: addressing additional layers of complexity. Clin. Pharmacol. Ther. 113, 957-959 (2023).