Editorial: Real world evidence, outcome research and healthcare management improvement through real world data (RWD).
Ontology highlight
ABSTRACT:
SUBMITTER: Varela-Rodriguez C
PROVIDER: S-EPMC9846327 | biostudies-literature | 2022
REPOSITORIES: biostudies-literature
Publications
Editorial: Real world evidence, outcome research and healthcare management improvement through real world data (RWD).
Varela-Rodríguez Carolina C Rosillo-Ramirez Nicolás N Rubio-Valladolid Gabriel G Ruiz-López Pedro P
Frontiers in public health 20230104
Similar Datasets
Project description:The ASH Research Collaborative is a nonprofit organization established through the American Society of Hematology's commitment to patients with hematologic conditions and the science that informs clinical care and future therapies. The ASH Research Collaborative houses 2 major initiatives: (1) the Data Hub and (2) the Clinical Trials Network (CTN). The Data Hub is a program for hematologic diseases in which networks of clinical care delivery sites are developed in specific disease areas, with individual patient data contributed through electronic health record (EHR) integration, direct data entry through electronic data capture, and external data sources. Disease-specific data models are constructed so that data can be assembled into analytic datasets and used to enhance clinical care through dashboards and other mechanisms. Initial models have been built in multiple myeloma (MM) and sickle cell disease (SCD) using the Observational Medical Outcomes Partnership (OMOP) Common Data Model (CDM) and Fast Healthcare Interoperability Resources (FHIR) standards. The Data Hub also provides a framework for development of disease-specific learning communities (LC) and testing of health care delivery strategies. The ASH Research Collaborative SCD CTN is a clinical trials accelerator that creates efficiencies in the execution of multicenter clinical trials and has been initially developed for SCD. Both components are operational, with the Data Hub actively aggregating source data and the SCD CTN reviewing study candidates. This manuscript describes processes involved in developing core features of the ASH Research Collaborative to inform the stakeholder community in preparation for expansion to additional disease areas.
| S-EPMC9153041 | biostudies-literature
Project description:BackgroundDigital technologies are transforming the health care system. A large part of information is generated as real-world data (RWD). Data from electronic health records and digital biomarkers have the potential to reveal associations between the benefits and adverse events of medicines, establish new patient-stratification principles, expose unknown disease correlations, and inform on preventive measures. The impact for health care payers and providers, the biopharmaceutical industry, and governments is massive in terms of health outcomes, quality of care, and cost. However, a framework to assess the preliminary quality of RWD is missing, thus hindering the conduct of population-based observational studies to support regulatory decision-making and real-world evidence.ObjectiveTo address the need to qualify RWD, we aimed to build a web application as a tool to translate characterization of some quality parameters of RWD into a metric and propose a standard framework for evaluating the quality of the RWD.MethodsThe RWD-Cockpit systematically scores data sets based on proposed quality metrics and customizable variables chosen by the user. Sleep RWD generated de novo and publicly available data sets were used to validate the usability and applicability of the web application. The RWD quality score is based on the evaluation of 7 variables: manageability specifies access and publication status; complexity defines univariate, multivariate, and longitudinal data; sample size indicates the size of the sample or samples; privacy and liability stipulates privacy rules; accessibility specifies how the data set can be accessed and to what granularity; periodicity specifies how often the data set is updated; and standardization specifies whether the data set adheres to any specific technical or metadata standard. These variables are associated with several descriptors that define specific characteristics of the data set.ResultsTo address the need to qualify RWD, we built the RWD-Cockpit web application, which proposes a framework and applies a common standard for a preliminary evaluation of RWD quality across data sets-molecular, phenotypical, and social-and proposes a standard that can be further personalized by the community retaining an internal standard. Applied to 2 different case studies-de novo-generated sleep data and publicly available data sets-the RWD-Cockpit could identify and provide researchers with variables that might increase quality.ConclusionsThe results from the application of the framework of RWD metrics implemented in the RWD-Cockpit application suggests that multiple data sets can be preliminarily evaluated in terms of quality using the proposed metrics. The output scores-quality identifiers-provide a first quality assessment for the use of RWD. Although extensive challenges remain to be addressed to set RWD quality standards, our proposal can serve as an initial blueprint for community efforts in the characterization of RWD quality for regulated settings.
| S-EPMC9627468 | biostudies-literature
Project description:BackgroundThe availability of high fidelity electronic health record (EHR) data is a hallmark of the learning health care system. Washington State's Surgical Care Outcomes and Assessment Program (SCOAP) is a network of hospitals participating in quality improvement (QI) registries wherein data are manually abstracted from EHRs. To create the Comparative Effectiveness Research and Translation Network (CERTAIN), we semi-automated SCOAP data abstraction using a centralized federated data model, created a central data repository (CDR), and assessed whether these data could be used as real world evidence for QI and research.ObjectivesDescribe the validation processes and complexities involved and lessons learned.MethodsInvestigators installed a commercial CDR to retrieve and store data from disparate EHRs. Manual and automated abstraction systems were conducted in parallel (10/2012-7/2013) and validated in three phases using the EHR as the gold standard: 1) ingestion, 2) standardization, and 3) concordance of automated versus manually abstracted cases. Information retrieval statistics were calculated.ResultsFour unaffiliated health systems provided data. Between 6 and 15 percent of data elements were abstracted: 51 to 86 percent from structured data; the remainder using natural language processing (NLP). In phase 1, data ingestion from 12 out of 20 feeds reached 95 percent accuracy. In phase 2, 55 percent of structured data elements performed with 96 to 100 percent accuracy; NLP with 89 to 91 percent accuracy. In phase 3, concordance ranged from 69 to 89 percent. Information retrieval statistics were consistently above 90 percent.ConclusionsSemi-automated data abstraction may be useful, although raw data collected as a byproduct of health care delivery is not immediately available for use as real world evidence. New approaches to gathering and analyzing extant data are required.
| S-EPMC5983060 | biostudies-literature
Project description:BackgroundTreatment for pediatric psoriasis is challenging because of the lack of real-world evidence, especially for biological therapies.ObjectivesThis study evaluated the efficacy and safety of biologics in children with psoriasis based on real-world evidence.MethodsPediatric psoriasis patients aged <18 years who were treated with biologics in our hospital (2020-2022) were prospectively analyzed. Patients treated with adalimumab, secukinumab, or ixekizumab were followed up for at least 16 weeks, and 22 of 38 patients completed the 52-week observation period. Dermatologist raters were blinded to ensure the reliability of the PASI, BSA, and PGA score assessments. PASI 75 or PGA 0/1 at week 12 represented an efficient indicator.ResultsThirty-eight patients (20 males and 18 females; median age, 12.6 ± 4.1 years) were enrolled, and none were lost to follow-up. All participants were diagnosed with psoriasis, including plaque psoriasis (n = 36), nail psoriasis (n = 1), and pustular psoriasis (n = 1). Within 12 weeks, all patients achieved scores above PASI 75 and PGA 0/1. The average time to reach PASI 75 was 4.3 ± 2.0, 3.2 ± 1.8, and 2.4 ± 0.4 weeks in patients using adalimumab, secukinumab, and ixekizumab, respectively, and, 27.2% (3/11), 86.4% (19/22), and 75.0% (3/4) of these patients achieved PASI 100 at week 12, respectively. Moreover, 18 of 20 patients with plaque psoriasis maintained ≥PASI 75 after 52 weeks. The most commonly reported adverse effect was upper respiratory tract infection, and no severe adverse effects were reported.ConclusionsOur real-world data demonstrated the safety and effectiveness of adalimumab, secukinumab, and ixekizumab in children with psoriasis.
| S-EPMC9490006 | biostudies-literature
Project description:BackgroundThere is significant unmet need for effective and efficiently delivered care for people with Parkinson's disease (PwP). We undertook a service improvement initiative to co-develop and implement a new care pathway, Home Based Care (HBC), based on supported self-management, remote monitoring and the ability to trigger a healthcare contact when needed.ObjectiveTo evaluate feasibility, acceptability and safety of Home Based Care.MethodsWe evaluated data from the first 100 patients on HBC for 6 months. Patient monitoring, performed at baseline and 6-monthly, comprised motor (MDS-UPDRS II and accelerometer), non-motor (NMSQ, PDSS-2, HADS) and quality of life (PDQ) measures. Care quality was audited against Parkinson's UK national audit standards. Process measures captured feasibility. Acceptability was assessed using a mixed-methods approach comprising questionnaires and semi-structured interviews.ResultsBetween October 2019 and January 2021, 108 PwP were enrolled onto HBC, with data from 100 being available at 6 months. Over 90% of all questionnaires were returned, 97% were complete or had < 3 missing items. Reporting and communications occurred within agreed timeframes. Compared with baseline, after 6m on HBC, PD symptoms were stable; more PwP felt listened to (90% vs. 79%) and able to seek help (79% vs. 68%). HBC met 93% of national audit criteria. Key themes from the interviews included autonomy and empowerment.ConclusionsWe have demonstrated acceptability, feasibility and safety of our novel remotely delivered Parkinson's care pathway. Ensuring scalability will widen its reach and realize its benefits for underserved communities, enabling formal comparisons with standard care and cost-effectiveness evaluation.
| S-EPMC10836560 | biostudies-literature
Project description:Aim: Comparative effectiveness research (CER) is essential for making informed decisions about drug access. It provides insights into the effectiveness and safety of new drugs compared with existing treatments, thereby guiding better healthcare decisions and ensuring that new therapies meet the real-world needs of patients and healthcare systems. Objective: To provide a tool that assists analysts and decision-makers in identifying the most suitable analytical approach for answering a CER question, given specific data availability contexts. Methods: A systematic literature review of the scientific literature was performed and existing regulatory and health technology assessment (HTA) guidance were evaluated to identify and compare recommendations and best practices. Based on this review a methods flowchart that synthesizes current practices and requirements was proposed. Results: The review did not find any papers that clearly identified the most appropriate analytical approach for answering CER questions under various conditions. Therefore, a methods flowchart was designed to inform analyst and decision makers choices starting from a well-defined scientific question. Conclusion: The proposed methods flowchart offers clear guidance on CER methodologies across a range of settings and research needs. It begins with a well-defined research question and considers multiple feasibility aspects related to CER. This tool aims to standardize methods, ensure rigorous and consistent research quality and promote a culture of evidence-based decision-making in healthcare.
| S-EPMC11609978 | biostudies-literature
Project description:The retrospective, observational RWD-ACROSS study analyzed disease characteristics, systemic treatment, and survival in patients with metastatic colorectal cancer (mCRC) in Spain. In total, 2002 patients were enrolled (mean age 65.3 years; 62.7% male). Overall median overall survival (OS) was 26.72 months, and was longer in patients with left-sided tumors (28.85 vs. 21.04 months (right-sided tumors); p < 0.0001) and in patients receiving first-line anti-epidermal growth factor receptor (EGFR) treatment (31.21 vs. 26.75 (anti-vascular endothelial growth factor (VEGF) treatment) and 24.45 months (chemotherapy); p = 0.002). Overall median progression-free survival (PFS) was 10.72 months and was longer in patients with left-sided tumors (11.24 vs. 9.31 months (right-sided tumors); p < 0.0001), and in patients receiving either first-line anti-EGFR or anti-VEGF (12.13 and 12.00 vs. 8.98 months (chemotherapy); p < 0.001). PFS was longer with anti-VEGF treatment in patients with right-sided tumors and wild-type RAS (11.24 vs. 8.78 (anti-EGFR) and 7.83 months (chemotherapy); p = 0.025). Both anti-EGFR and anti-VEGF produced longer PFS in patients with left-sided tumors and wild-type RAS than chemotherapy alone (12.39 and 13.14 vs. 9.83 months; p = 0.011). In patients with left-sided tumors and mutant RAS, anti-VEGF produced a longer PFS than chemotherapy alone (12.36 vs. 9.34 months; p = 0.001). In Spain, wild-type RAS or left-sided mCRC tumors are predictive of longer survival times.
| S-EPMC10526223 | biostudies-literature
Project description:Due to the COVID-19 pandemic, all countries with a global mobilization started to produce and authorize vaccines, prioritizing healthcare professionals (HCPs) to reduce transmission. The aim of this study was to assess post-vaccination infections' occurrence among HCPs and their correlation with symptom onset. A retrospective cohort study was carried out in the Campania Region from December 2020 to April 2021. Data were retrieved from the Regional Health Information System of the Campania Region (Sinfonia). The study cohort included subjects that had all received at least one vaccine dose. Risk ratios (RRs) adjusted for age and sex (95% confidence intervals) were performed to assess differences in the prevalence between HCPs who tested positive or negative for COVID-19. Univariate and multivariate logistic regression models were used to evaluate the association between symptoms and vaccination status. Findings revealed that HCPs had a lower risk of contracting COVID-19 after receiving at least one vaccine dose, and this risk decreased with age. Furthermore, not having full vaccination coverage may predict a severe/critical evolution of the disease. This study provides a snapshot of the initial state of the Italian vaccination campaign on HCPs. A surveillance approach using Big Data matched to clinical conditions could offer a real analysis in the categorization of subjects most at risk.
| S-EPMC9024651 | biostudies-literature
Project description:Evidence-based population interventions rely on intervention testing (efficacy and effectiveness trials) to determine what works to improve public health. We investigated the characteristics of real-world public health interventions to address obesity and explored the extent to which research testing was undertaken prior to scale-up. We identified 90 population health interventions targeting physical activity, nutrition or obesity-related health behaviours and collected publicly available information on their key characteristics and outcomes. We then assessed the differences between interventions that followed a research pathway and those that did not. Two-thirds (n = 60) of the interventions were reported as having followed a research pathway. Univariate logistic regression analysis revealed that these interventions were more likely to be health education interventions [odds ratio (OR): 5.56; 95% confidence interval (CI): 1.38-22.38], developed by research institutes (OR: 12.81; 95% CI: 3.47-47.34), delivered in North America (OR: 4.13; 95% CI: 1.61-10.62), and less likely to be owned (OR: 0.35; 95% CI: 0.14-0.88) or funded by government organizations (OR: 0.37; 95% CI: 0.14-0.95). Interventions that followed a research pathway were nearly three times more likely to have a positive impact on population health (OR: 2.72; 95% CI: 1.04-7.14). Interventions that followed a research pathway to scale-up were no more likely to be sustained longer than those that did not. Differences exist across real-world interventions between those that follow a research pathway to population-scale delivery and those that do not, regarding organizational and environmental context. A key benefit of research pathway to scale-up is the impact it has on health outcomes.
| S-EPMC11419317 | biostudies-literature
Project description:Various real-world data (RWD) sources have emerged in China with the intention of generating real-world evidence (RWE) that can be used in clinical and regulatory decision-making. Despite these efforts, significant barriers remain that hinder high-quality healthcare research. A workshop with 30 representatives from healthcare research agencies, technology companies focused on healthcare big data and pharmaceutical companies was held in December 2020 to identify strategies to overcome the barriers associated with the usability and quality of RWD in China. Across all sectors, examples of barriers identified included inconsistencies in terminology and non-standardised coding practices; the absence of longitudinal data; the absence of transparent data processing and validation practices; and the inability to access and share RWD. While cutting-edge technological innovations and data solutions provided powerful tools, the development of collaborative and synergistic research networks across multiple stakeholders is key to generate accessible, high-quality RWD in China. RWD has the potential to provide clinical, regulatory and reimbursement decision-makers with critical insights that can improve healthcare delivery in China. However, barriers to its access, collection and use must be addressed to generate RWE to guide healthcare stakeholders.
| S-EPMC9345036 | biostudies-literature