Abstract
The United States (US) Food and Drug Administration (FDA) Investigational New Drug (IND) Final Rule (US FDA, Final rule: Investigational new drug safety reporting requirements for human drug and biological products and safety reporting requirements for bioavailability and bioequivalence studies in humans, 2010) applies to all human drugs and biological products being studied under an IND. The Final Rule specifies that a sponsor must file an IND safety report for any Suspected Unexpected Serious Adverse Reaction (SUSAR) of a medicinal product being investigated. To make a proper SUSAR classification, sponsors need to go beyond conventional Data Monitoring Committees (DMCs) with an interdisciplinary effort, using all relevant data (including data outside clinical trials), to make judgments on the possibility of serious adverse events being caused by the study drug—rather than the underlying condition of the patient or a concomitant therapy. Ball et al. (Ball et al. in Ther Innov Regul Sci 55:705–716, 2021) have reported on how the Final Rule has been implemented by large pharmaceutical companies. This paper explores the experiences of small sponsor companies regarding the Final Rule, to understand the current challenges that they have been facing to meet aggregate IND safety reporting requirements.
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Acknowledgements
On behalf of the Drug Information Association (DIA)-American Statistical Association (ASA) Interdisciplinary Safety Evaluation (DAISE) scientific working group, which is part of the ASA Biopharm Safety Monitoring working group (with an official public private partnership with the US FDA), we targeted 37 small/medium-sized sponsors for participation in a survey. We would like to thank the 21 respondents for participating in this survey so that their experiences could be used to help other sponsors, as well as regulators at the US FDA (and potentially sponsors and regulatory authorities in other regions), to understand the current challenges that smaller sponsors have been facing to meet aggregate IND safety reporting requirements and, in particular, the 2021 draft guidance: Sponsor Responsibilities—Safety Reporting Requirements and Safety Assessment for IND and BA/BE Studies. We would also like to thank and recognize Jacqueline Corrigan-Curay for her engagement with the survey and review of the paper.
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GB, JB, and SS are independent consultants and could benefit from companies implementing the ASAP process. Otherwise, none of the authors have a financial or personal relationship with a third party whose interests could be positively or negatively influenced by the article’s content.
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Ball, G., Li, M., Buchanan, J. et al. Aggregate IND Safety Reporting for Smaller Companies and Programs. Ther Innov Regul Sci 58, 368–379 (2024). https://doi.org/10.1007/s43441-023-00605-2
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DOI: https://doi.org/10.1007/s43441-023-00605-2