Bmc Med Res Methodol
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Bmc Med Res Methodol · Jul 2016
LetterEFSPI/PSI working group on data sharing: accessing and working with pharmaceutical clinical trial patient level datasets - a primer for academic researchers.
Access to patient level datasets from clinical trial sponsors continues to be an important topic for the Pharmaceutical Industry as well as academic institutions and researchers. How to make access to patient level data actually happen raises many questions from the perspective of the researcher. ⋯ Whilst the benefits and value of patient level data sharing have yet to be fully realised, we hope that the information outlined in this article will encourage researchers to consider accessing and re-using clinical trial data to support their research questions.
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Greater transparency, including sharing of patient-level data for further research, is an increasingly important topic for organisations who sponsor, fund and conduct clinical trials. This is a major paradigm shift with the aim of maximising the value of patient-level data from clinical trials for the benefit of future patients and society. We consider the analysis of shared clinical trial data in three broad categories: (1) reanalysis - further investigation of the efficacy and safety of the randomized intervention, (2) meta-analysis, and (3) supplemental analysis for a research question that is not directly assessing the randomized intervention. ⋯ In order to support appropriate interpretation and limit the risk of misleading findings, analysis of shared clinical trial data should have a pre-specified analysis plan. However, it is not generally possible to limit bias and control multiplicity to the extent that is possible in the original trial design, conduct and analysis, and this should be acknowledged and taken into account when interpreting results. We highlight a number of areas where specific considerations arise in planning, conducting, interpreting and reporting analyses of shared clinical trial data. A key issue is that that these analyses essentially share many of the limitations of any post hoc analyses beyond the original specified analyses. The use of individual patient data in meta-analysis can provide increased precision and reduce bias. Supplemental analyses are subject to many of the same issues that arise in broader epidemiological analyses. Specific discussion topics are addressed within each of these areas. Increased provision of patient-level data from industry and academic-led clinical trials for secondary research can benefit future patients and society. Responsible data sharing, including transparency of the research objectives, analysis plans and of the results will support appropriate interpretation and help to address the risk of misleading results and avoid unfounded health scares.
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Bmc Med Res Methodol · Jun 2016
ReviewStepped wedge cluster randomised trials: a review of the statistical methodology used and available.
Previous reviews have focussed on the rationale for employing the stepped wedge design (SWD), the areas of research to which the design has been applied and the general characteristics of the design. However these did not focus on the statistical methods nor addressed the appropriateness of sample size methods used.This was a review of the literature of the statistical methodology used in stepped wedge cluster randomised trials. ⋯ Many studies which employ the stepped wedge design have few clusters but use methods of analysis which may require more clusters for unbiased and efficient intervention effect estimates. There is the need for research on the minimum number of clusters required for both types of stepped wedge design. Researchers should distinguish in the sample size calculation between cohort and cross sectional stepped wedge designs. Further research is needed on the effect of adjusting for the potential confounding of time on the study power.
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Bmc Med Res Methodol · Jun 2016
Modeling the oxygen uptake kinetics during exercise testing of patients with chronic obstructive pulmonary diseases using nonlinear mixed models.
The six-minute walk test (6MWT) is commonly used to quantify exercise capacity in patients with several cardio-pulmonary diseases. Oxygen uptake ([Formula: see text]O2) kinetics during 6MWT typically follow 3 distinct phases (rest, exercise, recovery) that can be modeled by nonlinear regression. Simultaneous modeling of multiple kinetics requires nonlinear mixed models methodology. To the best of our knowledge, no such curve-fitting approach has been used to analyze multiple [Formula: see text]O2 kinetics in both research and clinical practice so far. ⋯ Hereby we demonstrate the novelty and usefulness of this methodology in the context of physiological exercise testing.
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Bmc Med Res Methodol · May 2016
Using the modified Delphi method to establish clinical consensus for the diagnosis and treatment of patients with rotator cuff pathology.
Patients presenting to the healthcare system with rotator cuff pathology do not always receive high quality care. High quality care occurs when a patient receives care that is accessible, appropriate, acceptable, effective, efficient, and safe. The aim of this study was twofold: 1) to develop a clinical pathway algorithm that sets forth a stepwise process for making decisions about the diagnosis and treatment of rotator cuff pathology presenting to primary, secondary, and tertiary healthcare settings; and 2) to establish clinical practice guidelines for the diagnosis and treatment of rotator cuff pathology to inform decision-making processes within the algorithm. ⋯ This consensus guideline will help to standardize care, provide guidance on the diagnosis and treatment of rotator cuff pathology, and assist in clinical decision-making for all healthcare professionals.