Abstract
Purpose
The Pediatric Research Equity Act (PREA) gives the US Food and Drug Administration (FDA) authority to require pediatric studies for drug and biologics products under certain circumstances and to waive this requirement in some, or all, pediatric ages. When studies are waived for safety, PREA stipulates the safety issue must be described in labeling. This study assessed the rate of including waiver-related safety information in labeling.
Methods
FDA databases were reviewed to determine the number of safety-related pediatric study waivers and issued from December 2003 through August 2020, and corresponding labeling to establish when relevant safety information was included. Descriptive comparisons were conducted across Cohort 1: December 2003–2007, Cohort 2: 2008–2011, Cohort 3: 2012–2015, and Cohort 4: 2016-August 2020.
Results
One hundred sixteen safety waivers were issued [Cohort 1 (n = 1); Cohort 2 (n = 38), Cohort 3 (n = 37), and Cohort 4 (n = 40)] for 84 unique drugs or biologics. Most (106 of 116; 91%) waiver-related safety issues were described in labeling [Cohort 1 (1 of 1), Cohort 2 (33 of 38), Cohort 3 (33 of 37), and Cohort 4 (39 of 40)]. Safety waivers were most common in patients ≤ 17 years (n = 40) and least common in patients ≤ 6 months (n = 15). Products for infections (n = 32) were the most common group receiving safety waivers; 17 for non-antiviral anti-infective products including treatments for dermatologic infestations/infections, and 15 for antiviral products.
Conclusion
The data confirm that FDA consistently describes waiver-related safety information in drug/biologic product labeling since the inception of PREA in December of 2003.
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Data Availability
Data obtained from DARRTS is not publically available.
References
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https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm. Accessed 3 Jan 2023.
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EH drafted the research proposal. MB and DPB created the primary dataset. JK and EH analyzed the data, drafted the initial manuscript, and revised the manuscript. YM, JWM, JJA, and LY reviewed and revised the manuscript. All authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work.
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Krastein, J., Pica-Branco, D., Bacho, M.A. et al. Evaluation of FDA Labeling Changes Related to PREA Safety–Waivers. Ther Innov Regul Sci 57, 1099–1103 (2023). https://doi.org/10.1007/s43441-023-00551-z
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DOI: https://doi.org/10.1007/s43441-023-00551-z