Real-World Evidence Generation
Kelly H. Zou, Lobna A. Salem, Amrit Ray in Real-World Evidence in a Patient-Centric Digital Era, 2023
In contrast, observational study is a type of study in which individuals are observed or certain outcomes are measured (National Cancer Instittue, 2022). No attempt is made to affect the outcome (for example, no treatment is given.) As such, observational data are the essence of “real world.” Observational studies are non-experimental in nature, and thus their role and validity has been a controversial topic in the literature (Collins and Bowman 2020). Nonetheless, observation-based studies can suggest important areas for RCTs, hypothesis generation or clarify our understanding of patient experience. They can do so by utilizing various designs including case report or case series, ecologic, cross-sectional (i.e., a prevalence study), case-control and cohort studies (Kumar and Khan 2014).
How to conduct observational studies
Felicity Goodyear-Smith, Bob Mash in How to Do Primary Care Research, 2018
Observational studies are an increasingly popular type of investigation to gather data, information and evidence on a particular research question in almost all quantitative domains of primary care research. Areas of application include studies on programme and policy evaluation, field studies and surveys obtaining representative or purposive sample data from target populations. Due to the rapid advancements in information technology, recent observational studies are employing more and more administrative databases and electronic medical or health record data (see also Chapter 8 on the use of big data). Observational studies, as the name suggests, involve acquiring information (data) based on observation without intervening, and are therefore also referred to as non-interventional studies. In other words, observational studies allow investigation of various aspects of a research question under real-life conditions, without any external modification of the processes or procedures that relate to the population under study.
Combination Antimicrobial Therapy for Gram-Negative Infections: What Is the Evidence?
Robert C. Owens, Lautenbach Ebbing in Antimicrobial Resistance, 2007
However, the importance of monotherapy versus combination therapy for P. aeruginosa infections is controversial, whether for empiric therapy, or definitive treatment. RCTs of Gram-negative infections have too few cases of P. aeruginosa to allow a statistically robust analysis of the relative value of combination versus monotherapy. This was shown in a review of 10 randomized trials of antimicrobial therapy in patients with cancer and neutropenia. In this analysis, only 90 of a total of 909 episodes of bacteremia were caused by Pseudomonas species. Therefore, observational studies are important. Many have problems with selection bias, such as differences in severity of illness and comorbidities among patients receiving one or the other type of therapy. Other major shortcomings include lack of assessment of objective endpoints such as mortality. In addition, some do not account for the results of in vitro susceptibility testing in the definition of adequate therapy. Given these limitations, definitive conclusions regarding whether empiric or definitive combination therapy are superior to adequate monotherapy are difficult to make.
Flexible endoscopic evaluation of swallowing (FEES) to determine neurological intensive care patients’ oral diet
Published in International Journal of Speech-Language Pathology, 2021
Tobias Braun, Martin Juenemann, Maxime Viard, Marco Meyer, Iris Reuter, Stefan Mausbach, Johanna M. Doerr, Ingo Schirotzek, Mario Prosiegel, Patrick Schramm, Manfred Kaps, Christian Tanislav
To assess the value of FEES in neurological intensive care patients, we evaluated data from all patients in our department who received FEES during the course of 28 months. The study was designed as an observational study. We analysed the data for differences from FEES to clinical assessment and resulting change in oral diet, for differences in patients with and without dysphagia in regard to pneumonia, mortality, ventilation and length of stay in hospital and other outcome related parameters. Due to the fact that most of the patients on a neurological ICU suffer from stroke, we performed a subgroup analysis for this cohort. We also did the same analyses for patients with no oral feeding as compared to patients with at least small amounts of food or fluids. Patients without oral feeding are prone to malnutrition, even when treated in a hospital receiving parenteral or enteral feeding via nasogastric feeding tube (Butterworth, 1994; “Disease-related malnutrition and enteral nutrition therapy: a significant problem with a cost-effective solution,” 2010; Souza, Sturion, & Faintuch, 2015). Literature suggests a worse outcome in patients with no oral feeding, as they are predisposed to malnutrition (Newman et al., 2001; Schumann et al., 2012). Corresponding data from neurological ICU patients could not be identified at the time of data acquisition.
The prevalence of oropharyngeal dysphagia in patients with chronic obstructive pulmonary disease: a systematic review and meta-analysis
Published in Expert Review of Respiratory Medicine, 2022
Wenyan Li, Mingjing Gao, Jin Liu, Fengwa Zhang, Rongjing Yuan, Qingling Su, Yetong Wang, Yanhong Wang
Observational study (cross-sectional studies, case-control studies, and cohort studies).The subjects were patients with COPD (Diagnostic criteria for the GOLD guidelines [11]: the presence of progressive dyspnea, chronic cough, and expectoration; A history of exposure to risk factors; Pulmonary function tests: FEV1/FVC < 0.7 after inhalation of bronchodilators suggests continued airflow limitation, among other conditions that can cause continued airflow limitation.Assess for oropharyngeal dysphagia.
Gaps in evidence on treatment of male osteoporosis: a Research Agenda
Published in The Aging Male, 2023
Adam J. Rose, Susan L. Greenspan, Guneet K. Jasuja
It is therefore also important to consider how high-quality observational studies could inform clinical practice in our management of male osteoporosis. Observational studies can have important advantages – they can be conducted relatively quickly, they can have relatively low cost, they can involve large populations and thus have the potential to detect rare events, and they may in some ways be more generalizable than randomized trials – because their populations are more representative [74]. The advantages of randomized trials are also well-known, especially their ability to control for both observed and unobserved confounders, which has led to the justified emphasis on using them as the mainstay of clinical evidence to guide practice [75]. However, we think that high-quality observational studies of men with osteoporosis may be preferable to continuing to rely on evidence from clinical trials among women.
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