학술논문
Data sources and applied methods for paclitaxel safety signal discernment.
Document Type
Academic Journal
Author
Gressler LE; Office for Clinical Evidence and Analysis, United States Food and Drug Administration, Silver Spring, MD, United States.; Division of Pharmaceutical Evaluation and Policy, University of Arkansas for Medical Sciences, Little Rock, AR, United States.; Avila-Tang E; Office for Clinical Evidence and Analysis, United States Food and Drug Administration, Silver Spring, MD, United States.; Mao J; Department of Population Health Sciences, Weill Cornell Medicine, New York, NY, United States.; Avalos-Pacheco A; Applied Statistics Research Unit, Faculty of Mathematics and Geoinformation, TU Wien, Vienna, Austria.; Harvard-MIT Center for Regulatory Science, Harvard Medical School, Boston, MA, United States.; Shaya FT; School of Pharmacy, University of Maryland Baltimore, Baltimore, MD, United States.; Torosyan Y; Office for Clinical Evidence and Analysis, United States Food and Drug Administration, Silver Spring, MD, United States.; Division of Clinical Evidence and Analysis 3, United States Food and Drug Administration, Silver Spring, MD, United States.; Office of Product Evaluation and Quality, United States Food and Drug Administration, Silver Spring, MD, United States.; Liebeskind A; Office for Clinical Evidence and Analysis, United States Food and Drug Administration, Silver Spring, MD, United States.; Department of Population Health Sciences, Weill Cornell Medicine, New York, NY, United States.; Kinard M; Device Events, York, PA, United States.; Mack CD; IQVIA Real World Solutions, Research Triangle Park, Raleigh, NC, United States.; Department of Epidemiology, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC, United States.; Normand SL; Department of Health Care Policy, Harvard Medical School, Boston, MA, United States.; Department of Biostatistics, Harvard School of Public Health, Boston, MA, United States.; Ritchey ME; Med Tech Epi, Philadelphia, PA, United States.; Center for Pharmacoepidemiology and Treatment Science, Rutgers University, New Brunswick, NJ, United States.; Marinac-Dabic D; Office for Clinical Evidence and Analysis, United States Food and Drug Administration, Silver Spring, MD, United States.
Source
Publisher: Frontiers Media S.A Country of Publication: Switzerland NLM ID: 101653388 Publication Model: eCollection Cited Medium: Print ISSN: 2297-055X (Print) Linking ISSN: 2297055X NLM ISO Abbreviation: Front Cardiovasc Med Subsets: PubMed not MEDLINE
Subject
Language
English
ISSN
2297-055X
Abstract
Background: Following the identification of a late mortality signal, the Food and Drug Administration (FDA) convened an advisory panel that concluded that additional clinical study data are needed to comprehensively evaluate the late mortality signal observed with the use of drug-coated balloons (DCB) and drug-eluting stent (DES). The objective of this review is to (1) identify and summarize the existing clinical and cohort studies assessing paclitaxel-coated DCBs and DESs, (2) describe and determine the quality of the available data sources for the evaluation of these devices, and (3) present methodologies that can be leveraged for proper signal discernment within available data sources.
Methods: Studies and data sources were identified through comprehensive searches. original research studies, clinical trials, comparative studies, multicenter studies, and observational cohort studies written in the English language and published from January 2007 to November 2021, with a follow-up longer than 36 months, were included in the review. Data quality of available data sources identified was assessed in three groupings. Moreover, accepted data-driven methodologies that may help circumvent the limitations of the extracted studies and data sources were extracted and described.
Results: There were 39 studies and data sources identified. This included 19 randomized clinical trials, nine single-arm studies, eight registries, three administrative claims, and electronic health records. Methodologies focusing on the use of existing premarket clinical data, the incorporation of all contributed patient time, the use of aggregated data, approaches for individual-level data, machine learning and artificial intelligence approaches, Bayesian approaches, and the combination of various datasets were summarized.
Conclusion: Despite the multitude of available studies over the course of eleven years following the first clinical trial, the FDA-convened advisory panel found them insufficient for comprehensively assessing the late-mortality signal. High-quality data sources with the capabilities of employing advanced statistical methodologies are needed to detect potential safety signals in a timely manner and allow regulatory bodies to act quickly when a safety signal is detected.
Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
(© 2024 Gressler, Avila-Tang, Mao, Avalos-Pacheco, Shaya, Torosyan, Liebeskind, Kinard, Mack, Normand, Ritchey and Marinac-Dabic.)
Methods: Studies and data sources were identified through comprehensive searches. original research studies, clinical trials, comparative studies, multicenter studies, and observational cohort studies written in the English language and published from January 2007 to November 2021, with a follow-up longer than 36 months, were included in the review. Data quality of available data sources identified was assessed in three groupings. Moreover, accepted data-driven methodologies that may help circumvent the limitations of the extracted studies and data sources were extracted and described.
Results: There were 39 studies and data sources identified. This included 19 randomized clinical trials, nine single-arm studies, eight registries, three administrative claims, and electronic health records. Methodologies focusing on the use of existing premarket clinical data, the incorporation of all contributed patient time, the use of aggregated data, approaches for individual-level data, machine learning and artificial intelligence approaches, Bayesian approaches, and the combination of various datasets were summarized.
Conclusion: Despite the multitude of available studies over the course of eleven years following the first clinical trial, the FDA-convened advisory panel found them insufficient for comprehensively assessing the late-mortality signal. High-quality data sources with the capabilities of employing advanced statistical methodologies are needed to detect potential safety signals in a timely manner and allow regulatory bodies to act quickly when a safety signal is detected.
Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
(© 2024 Gressler, Avila-Tang, Mao, Avalos-Pacheco, Shaya, Torosyan, Liebeskind, Kinard, Mack, Normand, Ritchey and Marinac-Dabic.)