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Variation in Choice of Study Design: Findings from the Epidemiology Design Decision Inventory and Evaluation (EDDIE) Survey

Drug Safety volume 36, pages 15–25 (2013)Cite this article

Abstract

Background

Researchers using observational data to understand drug effects must make a number of analytic design choices that suit the characteristics of the data and the subject of the study. Review of the published literature suggests that there is a lack of consistency even when addressing the same research question in the same database.

Objective

To characterize the degree of similarity or difference in the method and analysis choices made by observational database research experts when presented with research study scenarios.

Research Design

On-line survey using research scenarios on drug-effect studies to capture method selection and analysis choices that follow a dependency branching based on response to key questions.

Subjects

Voluntary participants experienced in epidemiological study design solicited for participation through registration on the Observational Medical Outcomes Partnership website, membership in particular professional organizations, or links in relevant newsletters.

Measures

Description (proportion) of respondents selecting particular methods and making specific analysis choices based on individual drug-outcome scenario pairs. The number of questions/decisions differed based on stem questions of study design, time-at-risk, outcome definition, and comparator.

Results

There is little consistency across scenarios, by drug or by outcome of interest, in the decisions made for design and analyses in scenarios using large healthcare databases. The most consistent choice was the cohort study design but variability in the other critical decisions was common.

Conclusions

There is great variation among epidemiologists in the design and analytical choices that they make when implementing analyses in observational healthcare databases. These findings confirm that it will be important to generate empiric evidence to inform these decisions and to promote a better understanding of the impact of standardization on research implementation.

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Acknowledgments

The Observational Medical Outcomes Partnership is funded by the Foundation for the National Institutes of Health (FNIH) through generous contributions from the following: Abbott, Amgen Inc., AstraZeneca, Bayer Healthcare Pharmaceuticals, Inc., Bioden Idec, Bristol-Myers Squibb, Eli Lilly & Company, GlaxoSmithKline, Janssen Research and Development, Lundbeck, Inc., Merck & Co., Inc., Novartis Pharmaceuticals Corporation, Pfizer Inc, Pharmaceutical Research Manufacturers of America (PhRMA), Roche, Sanofi-aventis, Schering-Plough Corporation, and Takeda. Drs. Stang, Schuemie and Ryan are employees of Janssen Research and Development. Dr. Schuemie received a fellowship from the Office of Medical Policy, Center for Drug Evaluation and Research, Food and Drug Administration and has become an employee of Janssen Research & Development since completing this research.

Drs. Schuemie and Madigan have received a grant previously from FNIH. J. Marc Overhage, Abraham G. Hartzema, and Emily Welebob have no conflicts of interest to declare.

This article was published in a supplement sponsored by the Foundation for the National Institutes of Health (FNIH). The supplement was guest edited by Stephen J.W. Evans. It was peer reviewed by Olaf H. Klungel who received a small honorarium to cover out-of-pocket expenses. S.J.W.E has received travel funding from the FNIH to travel to the OMOP symposium and received a fee from FNIH for the review of a protocol for OMOP. O.H.K has received funding for the IMI–PROTECT project.from the Innovative Medicines Initiative Joint Undertaking (http://www.imi.europa.eu) under Grant Agreement no 115004, resources of which are composed of financial contribution from the European Union’s Seventh Framework Programme (FP7/2007–2013) and EFPIA companies’ in kind contribution.

Author information

Authors and Affiliations

  1. Janssen Research and Development LLC, Titusville, NJ, USA

    Paul E. Stang & Patrick B. Ryan

  2. 1125 Trenton-Harbourton Road, Room K30205, 200, Titusville, NJ, 08560, USA

    Paul E. Stang

  3. Siemens Health Services, Malvern, PA, USA

    J. Marc Overhage

  4. Department of Medical Informatics, Erasmus University Medical Center Rotterdam, Rotterdam, The Netherlands

    Martijn J. Schuemie

  5. College of Pharmacy, University of Florida, Gainesville, FL, USA

    Abraham G. Hartzema & Abraham G. Hartzema

  6. Observational Medical Outcomes Partnership, Foundation for the National Institutes of Health, Bethesda, MD, USA

    Paul E. Stang, Patrick B. Ryan, J. Marc Overhage, Martijn J. Schuemie & Emily Welebob

Authors
  1. Paul E. Stang
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  2. Patrick B. Ryan
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  3. J. Marc Overhage
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  4. Martijn J. Schuemie
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  5. Abraham G. Hartzema
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  6. Emily Welebob
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Corresponding author

Correspondence to Paul E. Stang.

Additional information

The OMOP research used data from Truven Health Analytics (formerly the Health Business of Thomson Reuters), and includes MarketScan® Research Databases, represented with MarketScan Lab Supplemental (MSLR, 1.2 m persons), MarketScan Medicare Supplemental Beneficiaries (MDCR, 4.6 m persons), MarketScan Multi-State Medicaid (MDCD, 10.8 m persons), MarketScan Commercial Claims and Encounters (CCAE, 46.5 m persons). Data also provided by Quintiles® Practice Research Database (formerly General Electric’s Electronic Health Record, 11.2 m persons) database. GE is an electronic health record database while the other four databases contain administrative claims data.

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Stang, P.E., Ryan, P.B., Overhage, J.M. et al. Variation in Choice of Study Design: Findings from the Epidemiology Design Decision Inventory and Evaluation (EDDIE) Survey. Drug Saf 36, 15–25 (2013). https://doi.org/10.1007/s40264-013-0103-1

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Keywords

  • Acute Myocardial Infarction
  • Propensity Score
  • Acute Liver Injury
  • Health Information Exchange
  • Analysis Choice