AUTHOR=Powell Michael , Koenecke Allison , Byrd James Brian , Nishimura Akihiko , Konig Maximilian F. , Xiong Ruoxuan , Mahmood Sadiqa , Mucaj Vera , Bettegowda Chetan , Rose Liam , Tamang Suzanne , Sacarny Adam , Caffo Brian , Athey Susan , Stuart Elizabeth A. , Vogelstein Joshua T. TITLE=Ten Rules for Conducting Retrospective Pharmacoepidemiological Analyses: Example COVID-19 Study JOURNAL=Frontiers in Pharmacology VOLUME=12 YEAR=2021 URL=https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2021.700776 DOI=10.3389/fphar.2021.700776 ISSN=1663-9812 ABSTRACT=

Since the beginning of the COVID-19 pandemic, pharmaceutical treatment hypotheses have abounded, each requiring careful evaluation. A randomized controlled trial generally provides the most credible evaluation of a treatment, but the efficiency and effectiveness of the trial depend on the existing evidence supporting the treatment. The researcher must therefore compile a body of evidence justifying the use of time and resources to further investigate a treatment hypothesis in a trial. An observational study can provide this evidence, but the lack of randomized exposure and the researcher’s inability to control treatment administration and data collection introduce significant challenges. A proper analysis of observational health care data thus requires contributions from experts in a diverse set of topics ranging from epidemiology and causal analysis to relevant medical specialties and data sources. Here we summarize these contributions as 10 rules that serve as an end-to-end introduction to retrospective pharmacoepidemiological analyses of observational health care data using a running example of a hypothetical COVID-19 study. A detailed supplement presents a practical how-to guide for following each rule. When carefully designed and properly executed, a retrospective pharmacoepidemiological analysis framed around these rules will inform the decisions of whether and how to investigate a treatment hypothesis in a randomized controlled trial. This work has important implications for any future pandemic by prescribing what we can and should do while the world waits for global vaccine distribution.