Triangulation of pharmacoepidemiology and laboratory science to tackle otic quinolone safety.


Journal

Basic & clinical pharmacology & toxicology
ISSN: 1742-7843
Titre abrégé: Basic Clin Pharmacol Toxicol
Pays: England
ID NLM: 101208422

Informations de publication

Date de publication:
Jan 2022
Historique:
revised: 15 09 2021
received: 07 06 2021
accepted: 04 10 2021
pubmed: 7 10 2021
medline: 25 3 2022
entrez: 6 10 2021
Statut: ppublish

Résumé

The scientific method requires studies with high internal and external validity. Though both are necessary, they do not go hand-in-hand: The more controlled a study is to enhance internal validity, the less applicable to real-world clinical care, and vice versa. In the many instances where evidence from clinical trials is not available, scientific inference must rely on more extreme approaches on this spectrum, such as mechanistic (limited generalizability/strong bias control) and real-world evidence (RWE) studies (higher generalizability/lesser bias control). Illustrate how triangulating mechanistic and RWE studies can enhance scientific inference by delivering the supporting evidence for both. We describe our research on an unexpected and highly unlikely drug safety issue: the risk of tympanic membrane (TM) perforations resulting from otic quinolone therapy. Tightly controlled laboratory studies using cell culture and rodent models were complemented with pharmacoepidemiological studies of real-world data to translate mechanistic findings and corroborate RWE. We present a cascade of mechanistic and RWE studies investigating fibroblast cytotoxicity, delayed healing of perforated TMs, and spontaneous TM perforations after otic quinolone exposure, all suggesting local tissue toxicity. Triangulation of mechanistic and RWE studies allowed incremental progress toward robust evidence on otic quinolone toxicity.

Sections du résumé

BACKGROUND BACKGROUND
The scientific method requires studies with high internal and external validity. Though both are necessary, they do not go hand-in-hand: The more controlled a study is to enhance internal validity, the less applicable to real-world clinical care, and vice versa. In the many instances where evidence from clinical trials is not available, scientific inference must rely on more extreme approaches on this spectrum, such as mechanistic (limited generalizability/strong bias control) and real-world evidence (RWE) studies (higher generalizability/lesser bias control).
OBJECTIVES OBJECTIVE
Illustrate how triangulating mechanistic and RWE studies can enhance scientific inference by delivering the supporting evidence for both.
METHODS METHODS
We describe our research on an unexpected and highly unlikely drug safety issue: the risk of tympanic membrane (TM) perforations resulting from otic quinolone therapy. Tightly controlled laboratory studies using cell culture and rodent models were complemented with pharmacoepidemiological studies of real-world data to translate mechanistic findings and corroborate RWE.
RESULTS RESULTS
We present a cascade of mechanistic and RWE studies investigating fibroblast cytotoxicity, delayed healing of perforated TMs, and spontaneous TM perforations after otic quinolone exposure, all suggesting local tissue toxicity.
CONCLUSION CONCLUSIONS
Triangulation of mechanistic and RWE studies allowed incremental progress toward robust evidence on otic quinolone toxicity.

Identifiants

pubmed: 34611995
doi: 10.1111/bcpt.13668
pmc: PMC9298360
doi:

Substances chimiques

Quinolones 0

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

75-80

Informations de copyright

© 2021 The Authors. Basic & Clinical Pharmacology & Toxicology published by John Wiley & Sons Ltd on behalf of Nordic Association for the Publication of BCPT (former Nordic Pharmacological Society).

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Auteurs

Almut G Winterstein (AG)

Pharmaceutical Outcomes and Policy, University of Florida, Gainesville, Florida, USA.
Center for Drug Evaluation and Safety, University of Florida, Gainesville, Florida, USA.
Epidemiology, University of Florida, Gainesville, Florida, USA.

Patrick J Antonelli (PJ)

Center for Drug Evaluation and Safety, University of Florida, Gainesville, Florida, USA.
Otolaryngology, University of Florida, Gainesville, Florida, USA.

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Classifications MeSH