STaRT-RWE: structured template for planning and reporting on the
                        implementation of real world evidence studies

Wang, Shirley; Pinheiro, Simone P.; Hua, Wei; Arlett, Peter; Uyama, Yoshiaki; Berlin, Jesse A.; Bartels, Dorothee B.; Kahler, Kristijan H.; Bessette, Lily G.; Schneeweiß, Sebastian

doi:10.1136/bmj.m4856

Cited by 125 publications

(97 citation statements)

References 23 publications

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“…Other efforts to advance evaluation of safety signals with real world data sources could also be considered. These assessments and evaluations must be made public, however, including specifying the safety outcome of interest in advance, to ensure their rigor and reliability 37. The FDA has indicated that it intends to further strengthen the Sentinel Initiative through user fee funds, increase public availability of the system, and enhance its ability to conduct analyses of specific safety issues 38.…”

Section: Discussionmentioning

confidence: 99%

Characterization and corroboration of safety signals identified from the US Food and Drug Administration Adverse Event Reporting System, 2008-19: cross sectional study

Dhodapkar

Shi²,

Ramachandran

et al. 2022

BMJ

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Objective To characterize potential drug safety signals identified from the US Food and Drug Administration (FDA) Adverse Event Reporting System (FAERS), from 2008 to 2019, to determine how often these signals resulted in regulatory action by the FDA and whether these actions were corroborated by published research findings or public assessments by the Sentinel Initiative. Design Cross sectional study. Setting USA. Population Safety signals identified from the FAERS and publicly reported by the FDA between 2008 and 2019; and review of the relevant literature published before and after safety signals were reported in 2014-15. Literature searches were performed in November 2019, Sentinel Initiative assessments were searched in December 2021, and data analysis was finalized in December 2021. Main outcome measures Safety signals and resulting regulatory actions; number and characteristics of published studies, including corroboration of regulatory action as evidenced by significant associations (or no associations) between the drug related to the signal and the adverse event. Results From 2008 to 2019, 603 potential safety signals identified from the FAERS were reported by the FDA (median 48 annually, interquartile range 41-61), of which 413 (68.5%) were resolved as of December 2021 (372 of 399 (93.2%) signals ≥3 years old were resolved). Among the resolved safety signals, 91 (22.0%) led to no regulatory action and 322 (78.0%) resulted in regulatory action, including 319 (77.2%) changes to drug labeling and 59 (14.3%) drug safety communications or other public communications from the FDA. For a subset of 82 potential safety signals reported in 2014-15, a literature search identified 1712 relevant publications; 1201 (70.2%) were case reports or case series. Among these 82 safety signals, 76 (92.7%) were resolved, of which relevant published research was identified for 57 (75.0%) signals and relevant Sentinel Initiative assessments for four (5.3%) signals. Regulatory actions by the FDA were corroborated by at least one relevant published research study for 17 of the 57 (29.8%) resolved safety signals; none of the relevant Sentinel Initiative assessments corroborated FDA regulatory action. Conclusions Most potential safety signals identified from the FAERS led to regulatory action by the FDA. Only a third of regulatory actions were corroborated by published research, however, and none by public assessments from the Sentinel Initiative. These findings suggest that either the FDA is taking regulatory actions based on evidence not made publicly available or more comprehensive safety evaluations might be needed when potential safety signals are identified.

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Section: Discussionmentioning

confidence: 99%

Characterization and corroboration of safety signals identified from the US Food and Drug Administration Adverse Event Reporting System, 2008-19: cross sectional study

Dhodapkar

Shi²,

Ramachandran

et al. 2022

BMJ

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“…These considerations may help researchers understand the strengths and limitations of different conceptual and operational approaches (e.g., selection of data sources, method of identifying medication) and contextualize their findings. These points are intended to complement considerations from existing rubrics, such as STaRT-RWE [6], the ISPE-ISPOR Consensus Document [7], RECORD-PE [8], and the ENCEPP Guide on Methodological Standards [9], which aid investigators in designing, conducting, reporting on, and comparing across studies.…”

Section: Discussionmentioning

confidence: 99%

Defining Medication Exposure When Analyzing Real-World Data

Clary

Lasky

et al. 2022

Preprint

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The conceptual definition of medication use includes the context under which the medication is being studied, the research concept of interest, the route of administration of interest, the dose of interest, and specifics regarding elements of timing and duration of medication use.• The operational definition of medication use includes a description of the data source, the approach used to identify the medication, how timing elements related to medication use were identified and Posted on

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“…This resulted in four eligible protocol templates; the European Medicines Agency (EMA) Heads of Medicines Agency Guideline on Good Pharmacovigilance Practices (GVP) Module VIII -post-authorizations safety studies (PASS) template, 20 ISPE's guidelines for good pharmacoepidemiology practice (GPP) section on protocol development, 24 National Evaluation System for health Technology (NEST) protocol guidance, 25 and the Structured Template and Reporting Tool for Real World Evidence (STaRT-RWE). 26 Section headings of the identified protocol templates were compared and mapped to each other, using the oldest guideline (EMA-GVP Module VIII-PASS) as the starting point (Table 1). The committee observed that at a conceptual level, the major elements of study design and analysis were already largely agreed upon and included in each of the templates.…”

Section: Identification and Comparison Of Protocol Templatesmentioning

confidence: 99%

“…Protocol templates that were not relevant for RWE studies that make secondary use of healthcare data or were not developed by international multi‐stakeholder groups were excluded. This resulted in four eligible protocol templates; the European Medicines Agency (EMA) Heads of Medicines Agency Guideline on Good Pharmacovigilance Practices (GVP) Module VIII ‐ post‐authorizations safety studies (PASS) template, 20 ISPE's guidelines for good pharmacoepidemiology practice (GPP) section on protocol development, 24 National Evaluation System for health Technology (NEST) protocol guidance, 25 and the Structured Template and Reporting Tool for Real World Evidence (STaRT‐RWE) 26 …”

Section: Introductionmentioning

confidence: 99%

HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real‐world evidence studies on treatment effects: A good practices report of a joint ISPE/ISPOR task force

Wang

Pottegård

Crown

et al. 2022

Pharmacoepidemiology and Drug

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STaRT-RWE: structured template for planning and reporting on the implementation of real world evidence studies

Cited by 125 publications

References 23 publications

Characterization and corroboration of safety signals identified from the US Food and Drug Administration Adverse Event Reporting System, 2008-19: cross sectional study

Characterization and corroboration of safety signals identified from the US Food and Drug Administration Adverse Event Reporting System, 2008-19: cross sectional study

Defining Medication Exposure When Analyzing Real-World Data

HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real‐world evidence studies on treatment effects: A good practices report of a joint ISPE/ISPOR task force

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