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      Common Problems, Common Data Model Solutions: Evidence Generation for Health Technology Assessment

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          Abstract

          There is growing interest in using observational data to assess the safety, effectiveness, and cost effectiveness of medical technologies, but operational, technical, and methodological challenges limit its more widespread use. Common data models and federated data networks offer a potential solution to many of these problems. The open-source Observational and Medical Outcomes Partnerships (OMOP) common data model standardises the structure, format, and terminologies of otherwise disparate datasets, enabling the execution of common analytical code across a federated data network in which only code and aggregate results are shared. While common data models are increasingly used in regulatory decision making, relatively little attention has been given to their use in health technology assessment (HTA). We show that the common data model has the potential to facilitate access to relevant data, enable multidatabase studies to enhance statistical power and transfer results across populations and settings to meet the needs of local HTA decision makers, and validate findings. The use of open-source and standardised analytics improves transparency and reduces coding errors, thereby increasing confidence in the results. Further engagement from the HTA community is required to inform the appropriate standards for mapping data to the common data model and to design tools that can support evidence generation and decision making.

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          ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions

          Non-randomised studies of the effects of interventions are critical to many areas of healthcare evaluation, but their results may be biased. It is therefore important to understand and appraise their strengths and weaknesses. We developed ROBINS-I (“Risk Of Bias In Non-randomised Studies - of Interventions”), a new tool for evaluating risk of bias in estimates of the comparative effectiveness (harm or benefit) of interventions from studies that did not use randomisation to allocate units (individuals or clusters of individuals) to comparison groups. The tool will be particularly useful to those undertaking systematic reviews that include non-randomised studies.
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            The FAIR Guiding Principles for scientific data management and stewardship

            There is an urgent need to improve the infrastructure supporting the reuse of scholarly data. A diverse set of stakeholders—representing academia, industry, funding agencies, and scholarly publishers—have come together to design and jointly endorse a concise and measureable set of principles that we refer to as the FAIR Data Principles. The intent is that these may act as a guideline for those wishing to enhance the reusability of their data holdings. Distinct from peer initiatives that focus on the human scholar, the FAIR Principles put specific emphasis on enhancing the ability of machines to automatically find and use the data, in addition to supporting its reuse by individuals. This Comment is the first formal publication of the FAIR Principles, and includes the rationale behind them, and some exemplar implementations in the community.
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              Bias and causal associations in observational research.

              Readers of medical literature need to consider two types of validity, internal and external. Internal validity means that the study measured what it set out to; external validity is the ability to generalise from the study to the reader's patients. With respect to internal validity, selection bias, information bias, and confounding are present to some degree in all observational research. Selection bias stems from an absence of comparability between groups being studied. Information bias results from incorrect determination of exposure, outcome, or both. The effect of information bias depends on its type. If information is gathered differently for one group than for another, bias results. By contrast, non-differential misclassification tends to obscure real differences. Confounding is a mixing or blurring of effects: a researcher attempts to relate an exposure to an outcome but actually measures the effect of a third factor (the confounding variable). Confounding can be controlled in several ways: restriction, matching, stratification, and more sophisticated multivariate techniques. If a reader cannot explain away study results on the basis of selection, information, or confounding bias, then chance might be another explanation. Chance should be examined last, however, since these biases can account for highly significant, though bogus results. Differentiation between spurious, indirect, and causal associations can be difficult. Criteria such as temporal sequence, strength and consistency of an association, and evidence of a dose-response effect lend support to a causal link.
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                Author and article information

                Contributors
                Jacoline.Bouvy@nice.org.uk
                Journal
                Pharmacoeconomics
                Pharmacoeconomics
                Pharmacoeconomics
                Springer International Publishing (Cham )
                1170-7690
                1179-2027
                18 December 2020
                18 December 2020
                : 1-11
                Affiliations
                [1 ]GRID grid.416710.5, ISNI 0000 0004 1794 1878, National Institute for Health and Care Excellence, ; London, United Kingdom
                [2 ]GRID grid.4991.5, ISNI 0000 0004 1936 8948, Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology, and Musculoskeletal Sciences (NDORMS), , University of Oxford, ; Oxford, UK
                [3 ]Fundació Institut Universitari per a la recerca a l’Atenció Primària de Salut Jordi Gol i Gurina (IDIAPJGol), Barcelona, Spain
                [4 ]Pfizer Inc, Oslo, Norway
                [5 ]GRID grid.419619.2, ISNI 0000 0004 0623 0341, Janssen Research and Development, ; Beerse, Belgium
                [6 ]GRID grid.5645.2, ISNI 000000040459992X, Department of Medical Informatics, , Erasmus University Medical Center, ; Rotterdam, The Netherlands
                Author information
                http://orcid.org/0000-0001-7298-3163
                Article
                981
                10.1007/s40273-020-00981-9
                7746423
                33336320
                36b7f073-7860-46c8-9c09-881b545831e3
                © The Author(s) 2020

                Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/.

                History
                : 5 November 2020
                Funding
                Funded by: FundRef http://dx.doi.org/10.13039/501100010767, Innovative Medicines Initiative;
                Award ID: 806968
                Categories
                Practical Application

                Economics of health & social care
                Economics of health & social care

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