Understanding variation in the results of real-world evidence studies that seem to address the same question

Wang, Shirley; Sreedhara, Sushama Kattinakere; Bessette, Lily G.; Schneeweiß, Sebastian

doi:10.1016/j.jclinepi.2022.08.012

Cited by 6 publications

(7 citation statements)

References 33 publications

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“…The result of the meta‐analysis of these three studies is in favor of an absence of risk of appendicitis 21 days after mRNA COVID‐19 vaccination but with a strong heterogeneity, making the unique role of sampling variance unlikely. Heterogeneity between observational studies addressing the same research question may occur from differences in data sources, set of inclusion/exclusion criteria, confounders, duration of baseline assessment, date of entry in the cohort, outcome and exposure risk algorithm and analytical strategies… 7 , 8 Our analysis suggests that the discrepancies between results of these studies is mainly linked to the choice of control groups. Comparator selection is cornerstone in observational studies to limit confounding by indication, leading to an imbalance between treatment groups in the baseline level of risk for the outcome of interest.…”

Section: Discussionmentioning

confidence: 99%

“…analysis choices for studies conducted in healthcare database is becoming feasible and could be one of the future direction to appraise the robustness of pharmacoepidemiological results. 7…”

Section: Discussionmentioning

confidence: 99%

“…Such sensitivity analyses must ideally be formalized a priori rationale to aid decision‐maker assessment and increase the utility of the findings 15 . Yet, recent studies have demonstrated that conducting hundreds or thousands of sensitivity analyses to evaluate the influence of design and analysis choices for studies conducted in healthcare database is becoming feasible and could be one of the future direction to appraise the robustness of pharmacoepidemiological results 7 …”

Section: Discussionmentioning

confidence: 99%

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Understanding the variability of pharmaco‐epidemiological studies assessing the risk of appendicitis with mRNA COVID‐19 vaccines

Jambon-Barbara

Bernardeau

Cracowski

et al. 2022

Pharmacoepidemiology and Drug

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Understanding the variability of pharmaco‐epidemiological studies assessing the risk of appendicitis with mRNA COVID‐19 vaccines

Jambon-Barbara

Bernardeau

Cracowski

et al. 2022

Pharmacoepidemiology and Drug

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“…Но если результат не воспроизводим, мало оснований для оценки его достоверности. Таким образом, достижение воспроизводимости является фундаментальным шагом для оценки достоверности исследования [6].…”

Section: введение введениеunclassified

“…Причинами невоспроизводимости могут быть: • неполная информация о деталях дизайна исследования и параметрах анализа; • внутренне противоречивая информация между текстом, таблицами, расчётными схемами и приложениями; • отклонения в результатах ввиду неполной информации о версии исходных данных [6].…”

Section: введение введениеunclassified

HARPER — protocol template for real-world trials for evaluating treatment efficacy

Mukhina

Baranova

Balykina

2023

MyRWD

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The International Society of Pharmacoepidemiology (ISPE) and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) have created a joint task force, including representatives of key international stakeholders, to create a coordinated protocol template for RWE studies that assess the effect of treatment and is designed to inform decision-making. The template is based on existing transparency efforts and includes the state-of-art ideas regarding the level of detail required to ensure reproducibility of the RWE study. The underlying principle was to achieve sufficient clarity regarding research data, design, analysis and implementation to achieve the three main objectives. Firstly, to help researchers carefully consider and document the choice and justification of the key parameters of the study, secondly, to facilitate decision-making by allowing reviewers to easily assess potential bias associated with the choice, and thirdly, to ensure research reproducibility.

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Tell me what you want, what you really really want: Estimands in observational pharmacoepidemiologic comparative effectiveness and safety studies

Luijken

Eekelen²,

Gardarsdóttir

et al. 2023

Pharmacoepidemiology and Drug

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Purpose: Ideally, the objectives of a pharmacoepidemiologic comparative effectiveness or safety study should dictate its design and data analysis. This paper discusses how defining an estimand is instrumental to this process. Methods:We applied the ICH-E9 (Statistical Principles for Clinical Trials) R1 addendum on estimandswhich originally focused on randomized trialsto three examples of observational pharmacoepidemiologic comparative effectiveness and safety studies. Five key elements specify the estimand: the population, contrasted treatments, endpoint, intercurrent events, and population-level summary measure.Results: Different estimands were defined for case studies representing three types of pharmacological treatments: (1) single-dose treatments using a case study about the effect of influenza vaccination versus no vaccination on mortality risk in an adult population of ≥60 years of age; (2) sustained-treatments using a case study about the effect of dipeptidyl peptidase 4 inhibitor versus glucagon-like peptide-1 agonist on hypoglycemia risk in treatment of uncontrolled diabetes; and (3) as needed treatments using a case study on the effect of nitroglycerin spray as-needed versus no nitroglycerin on syncope risk in treatment of stabile angina pectoris. Conclusions:The case studies illustrated that a seemingly clear research question can still be open to multiple interpretations. Defining an estimand ensures that the study targets a treatment effect that aligns with the treatment decision the study aims to inform. Estimand definitions further help to inform choices regarding study design and data-analysis and clarify how to interpret study findings.

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Understanding variation in the results of real-world evidence studies that seem to address the same question

Cited by 6 publications

References 33 publications

Understanding the variability of pharmaco‐epidemiological studies assessing the risk of appendicitis with mRNA COVID‐19 vaccines

Understanding the variability of pharmaco‐epidemiological studies assessing the risk of appendicitis with mRNA COVID‐19 vaccines

HARPER — protocol template for real-world trials for evaluating treatment efficacy

Tell me what you want, what you really really want: Estimands in observational pharmacoepidemiologic comparative effectiveness and safety studies

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