In recent years, real-world evidence data (RWD) and real-world evidence (RWE) have gained substantial attentions from healthcare practitioners and health authorities worldwide. In particular, the needs from regulatory bodies have promoted the production and use of real-world evidence. In the context of drug and device evaluation and regulation decisions, the pattern for using real world evidence may differ. This article aimed to discuss the potential uses of RWE for pre-approval clinical evaluation, post-approval monitoring and evaluation, and associated regulatory decisions, which may ultimately improve the production and use of RWE for regulatory decisions.
ObjectiveTo evaluate the safety, efficacy, and cost-effectiveness of different uses of oxidized regenerated cellulose (ORC) in video-assisted thoracoscopic surgery (VATS) for lung cancer resection to provide a reference for the selection, clinical use, and rational utilization of absorbable hemostatic materials. MethodsA retrospective analysis of relevant data from inpatients who underwent VATS for lung cancer resection at a tertiary hospital from July 2019 to January 2020 and from July 2020 to December 2020 was conducted. Patients were divided into two groups based on the use of ORC: 1) combined use group (ORC and collagen sponge) and 2) sole-use group (ORC). Safety, efficacy, and economic outcome indicators were compared between the two groups. ResultsThe main analysis included a total of 904 patients, with 466 in the combined use group and 438 in the sole-use group. Compared to the combined use group, the sole-use group had a significantly longer hospital stay, used fewer hemostatic drugs, had a lower average cost of hemostatic materials, and a lower median total hospitalization cost (P<0.05). No statistically significant difference was found between the two groups in terms of intraoperative blood loss volume, massive blood loss rate, perioperative transfusion rate, reoperation rate, postoperative 48-hour drainage volume, bloody drainage fluid rate, or postoperative laboratory test indicators. ConclusionThere was no significant difference in the safety or efficacy of VATS for lung cancer resection between the sole use of ORC and the combined use of ORC, but the sole use of ORC was associated with a lower cost of hemostatic materials and a lower total hospitalization cost. The sole use of hemostatic gauze in VATS for lung cancer resection may be a more cost-effective choice.
Observational studies based on real-world data are providing increasing amount of evidence for evaluating therapeutic outcomes, which is important for timely decision-making. Although time and costs for data collection could be saved using real-world data, it is significantly more complex to design real world researches with lower risk of bias. In order to enhance the validity of causal inference and to reduce potential risk of bias in real world studies, the Working Group of China Real world data and studies Alliance (China REAL) has formulated recommendations for designing observational studies to evaluate therapeutic outcomes based on real-world data. This guidance introduces design types commonly used in real world research; recommends key elements to consider in observational studies, including sample selection, specifying and allocating exposures, defining study entry and endpoints, and pre-designing statistical analysis protocols; and summarizes potential biases and corresponding control measures in real-world studies. These recommendations introduces key elements in designing observational studies using real-world data, for the purpose of improving the validity of causal inference. However, the application scope of these recommendations may be limited and warrant constant improvement.
ObjectiveTo explore whether education and management of medical care integration can improve asthma control. MethodsA prospective, 12-month, cohort study was undertaken in a real-world setting based on Australasian severe asthma network (ASAN). A total of 516 patients with stable asthma were consecutively recruited, who received education and management of medical care integration, and step-wise anti-asthma regimens determined by physicians’ standard practice. Furthermore, inhaled corticosteroid (ICS) adherence, lung function, asthma symptom control and exacerbation were assessed at 1, 3, 6, and 12 months. ResultsAt the end of 12 months, ICS adherence (47.7% vs. 81.5%, P<0.05), lung function, and asthma symptoms were assessed by asthma control text (ACT) [20 (16, 23) vs. 23 (21, 24), P<0.05], which were significantly improved in comparison to the status at baseline, and 86.0% of patients achieved total/well-controlled level of asthma. The exacerbation (14.2% vs. 36.2%, P<0.01) and hospitalizations (8.5% vs. 15.3%, P<0.01) because of asthma for the following year significantly decreased compared with those in the past year. The multivariate regression analysis indicated that poor ICS adherence (RR=1.52, 95%CI 1.02 to 2.25, P=0.039), depression symptoms (RR=1.19, 95%CI 1.05 to 1.34, P=0.007), and exacerbation during the past year (RR=2.81, 95%CI 1.49 to 5.27, P=0.001) were associated with an increased risk of future exacerbation. ConclusionIn a real-world setting, most of asthmatics achieve total/well-controlled asthma by education and management of medical care integration including shared decision-making between physicians and patients and step-wise anti-asthma regimens. ICS adherence and depression symptoms independently predict asthma exacerbations, and strengthening education and management of medical care integration, esp. psychological nursing, would improve asthma control levels.
Real-world data (RWD) in clinical research on specific categories of medical devices can generate sufficient quality evidence which will be used in decision making. This paper discusses the limitations of traditional randomized controlled trials in clinical research of medical devices, summarizes and analyses the applicable conditions of real-world evidence (RWE) for medical devices, interprets the new FDA guidance document on the characteristics of RWD for medical devices, in order to provide evidence for the use of RWE in medical devices in our country.
With the acceleration of global innovative drug development, selecting safe, effective, and cost-effective products from numerous drugs has posed new challenges for the decision-making process of medical insurance drug access and dynamic updating of insurance directory. Real-world data (RWD) provides a new perspective for evaluation of clinical and economic value of drugs, but there are still uncertainties regarding the scope, quality standards, and evidence categories of RWD that can be used. Based on the current status of domestic and international RWD supporting the assessment of the clinical and economic value of drugs, this paper, in collaboration with national RWD and healthcare experts, has developed the key considerations for using real-world data to evaluate the clinical and economic value of drugs. This paper first clarifies the scope of RWD that can be used to evaluate the clinical and economic value of drugs evaluate; secondly, provides specific requirements and guidance on data attribution, data governance, and quality standards for RWD; finally, summarizes the evidence categories of RWD supporting evaluate the clinical and economic value of drugs evaluate.
Randomized double-blind controlled trials (RCTs) conduct researches in carefully selected populations to ensure results of RCTs are unaffected by external disturbances and provide evidence of safety and efficacy. Real-world researches further help to understand the real world effects of new technologies in different medical environments after-market authorization. RCTs are the evidence foundation of real-world researches, and real-world researches provide valuable complement to RCTs. Medical insurance database is one of the most important database in real-world researches. Now, China's national medical insurance is entering a new era and transits from passive payment and compensation into a value-based strategic purchase mechanism for its insured population to buy the most cost-effective services. It is necessary to establish a mature, well-organized and value-based mechanism. The core of such mechanism is values, which is the price/performance ratio of innovative medicines and technologies rather than looking at the price solely. Demonstrating innovative drug value is an essential part of health care assessment. The authors argue that the assessment of the overall value of innovative technologies or medicines should include and based on the following four dimensions: clinical value, economic value, patient value and society value.
With the boom of information technology and data science, real-world evidence (RWE) which is produced using diverse real-world data (RWD) has become an important source for healthcare practice and policy decisions, such as regulatory and coverage decisions, guideline development, and disease management. The production of high-quality RWE requires not only complete, accurate and usable data, but also scientific and sound study designs and data analyses to enable the questions of interest to be reliably answered. In order to improve the quality of production and use of RWE, China REal world data and studies ALliance (ChinaREAL) has developed the first series of technical guidance for developing real-world data and subsequent studies. The efforts are ongoing which would ultimately inform better healthcare practice and policy decisions.
ObjectiveTo systematically review the difference in 30-day readmission rates among acute heart failure patients between real-world studies vs. randomized controlled trials (RCTs). MethodsPubMed, EMbase, The Cochrane Library, CNKI, CBM, VIP and WanFang Data databases were electronically searched to collect clinical studies on 30-day readmission rates in patients with acute heart failure from inception to April 12th, 2021. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies. Meta-analysis was then performed using Stata 16.0 software. ResultsA total of 33 real-world studies and 11 RCTs involving 106 722 subjects were included. The results of meta-analysis showed that the 30-day heart failure-related readmission rates in the real-world studies and RCTs were 10.8% (95%CI 9.3% to 12.3%) and 6.9% (95%CI 5.3% to 8.4%), respectively. The 30-day all-cause readmission rates in real-world studies and randomized controlled studies were 18.6% (95%CI 15.7% to 21.4%) and 14.2% (95%CI 12.0% to 16.3%), respectively. There were statistically significant differences between two kinds of studies (P<0.05). ConclusionsCurrent evidence suggests that the 30-day heart failure-related and all-cause readmission rates in patients of acute heart failure in real-world studies are significantly higher than those in patients of RCTs. Due to the limited quality and quantity of included studies, more high-quality studies are required to verify the above conclusions.
Real-world evidence represents critical evidence to support post-marketing drug monitoring, assessment and policy decisions, and has received extensive attentions. However, an explicit over-arching design and conceptual framework for this specific area is lacking. Divergent opinions on the production of real world evidence are often present among researchers; and understanding about their implications also differ among policy makers and evidence users. In this article, we have proposed, from the regulatory and clinical perspectives, a conceptual framework on the use of real world data for post-marketing drug studies, assessment and policy decisions.