With the real-world study (RWS) becoming a hotspot for clinical research, health data collected from routine clinical practice have gained increasing attention worldwide, particularly the data related to the off-label use of drugs, which have been at the forefront of clinical research in recent years. The guidance from the National Medical Products Administration has proposed that real-world evidence (RWE) can be an important consideration in supporting label expansions where randomized controlled trials are unfeasible. Nevertheless, how to use the RWE to support the approval of new or expanded indications remains unclear. This study aims to explore the structured process for the use of RWE in supporting label expansions of approved drugs, and to discuss the key considerations in such process by reviewing the documents from relevant regulatory agencies and publications from public databases, which can inform future directions for studies in this area.
The application of economic tools to evaluate the cost and health benefits and screen out more cost-effective drugs and technologies is an important measure to improve efficiency of medical resource allocation in China. Given the inherent differences between strict clinical trials and clinical routine practice, using trial-based economic evaluations to guide relevant medical decisions may lead to a certain risk of value deviation. Recent development of real-world data provides opportunities to assess the cost-effectiveness of drugs under the practical utilization, and has gradually become a new research hotspot. However, the complexity of the actual clinical environment also puts higher demands on researchers and decision makers to construct, understand and apply real-world evidence. In order to further prompt the normalization of economic evaluation based on real-world data and promote the scientific application of real-world evidence in medical and health decision-making, this project aims at the crucial issues including scope, research design and quality evaluation, to clarify the key considerations on the using of real-world evidence in medical decision-making. Combined with the international guidelines, the latest advancement of relevant research areas and the advice and opinions from multidisciplinary experts, we aim to provide technical references and guidance for researchers and decision makers, and to strengthen the evidence base of management policies.
Diabetic retinopathy (DR), which is a common complication of diabetic and the main cause of blindness, brings not only a heavy economic burden to society, but also seriously threatens to the patients’ quality of life. Clinical researches on the therapies of DR are active at present, but how to perform a good clinical research with scientific design should be considered with high priority. The randomized controlled trial (RCT) is considered to be the gold standard for evidence-based medicine, but RCT is not always perfect. Limitations still exist in certain circumstance and the conclusions from RCTs also need to be interpreted by an objective point of view before clinical practice. Real world study (RWS) bridges the gap between RCT and clinical practice, in which the data can be easily collected without much cost, and results might be obtained within a short period. However, RWS is also faced with the challenge of not having standardized data and being susceptible to confounding bias. The standardized single disease database for DR and propensity score matching method can provide a wide range of data sources and avoid of bias for RWS in DR.
ObjectiveTo evaluate changes in operational effectiveness after the implementation of ambulatory surgical management in pars plana vitrectomy (PPV). MethodsA retrospective clinical study. 17 528 surgeries in 10 895 eyes of 10 895 patients who underwent minimally invasive PPV on an ambulatory and/or inpatient basis at Tianjin Medical University Eye Hospital from August 2015 to June 2023 were included in this study. Among them, 5 346 eyes in 5 346 cases were male; 5 549 eyes in 5 549 cases were female. The age ranged from 0 to 95 years, with the mean age of (57.74±13.15) years. 6 381 surgeries in 3 615 eyes from August 2015 to December 2018 (the initial period of day surgery) were used as the control group; 11 147 surgeries in 7 280 eyes from January 2019 to June 2023 (the expanded period of day surgery) were used as the observation group. According to the management mode of ambulatory surgery, the observation group was subdivided into the decentralized management group (January 2019 to December 2020) and the centralized management group (January 2021 to June 2023), with 2 905 and 4 375 eyes and 4 646 and 6 501 surgeries, respectively. Changes in the percentage of day surgery, average hospitalization days, and average unplanned reoperation rate were compared. The Mann-Whitney U test was used to compare numerical variables between groups; the chi-square test or Fisher's exact test was used to compare categorical variables. ResultsThe number of cases of daytime PPV performed in the observation group and control group was 7 852 (70.44%, 7 852/11 147) and 24 (0.38%, 24/6 381) cases, respectively, and the average hospitalization days were 1 (1) and 5 (3) d. Compared with the control group, the observation group had a significantly higher percentage of day surgery (χ2=8 051.01) and a considerably lower mean hospitalization day (Z=4 536 844.50), and the differences were statistically significant (P＜0.000 1). The mean hospitalization days in the decentralized and centralized management groups were 2 (3) and 1 (0) d, respectively, and unplanned reoperations were 34 (0.73%, 34/4 646) and 171 (2.63%, 171/6 501) eyes, respectively. Compared with the decentralized management group, average hospitalization days was significantly lower (Z=1 436.94) and unplanned reoperation rate was significantly higher (χ2=54.10) were significantly lower in the centralized management group, both of which were statistically significant (P＜0.000 1). ConclusionPPV ambulatory management model can significantly reduce the average hospitalization day, but also results in higher rates of unplanned reoperations.
To enhance the quality and transparency of oncology real-world evidence studies, the European Society for Medical Oncology (ESMO) has developed the first specific reporting guidelines for oncology RWE studies in peer-reviewed journals "the ESMO Guidance for Reporting Oncology Real-World Evidence (GROW)". To facilitate readers understanding and application of these reporting standards, this article introduces and interprets the development process and main contents of the ESMO-GROW checklist.
Real-world data studies have experienced rapid development in recent years, however, misunderstandings persist. This paper aims to improve practice and promote standardization by updating the categorization of real-world data, proposing two conceptual frameworks for conducting real-world data studies, developing the concepts of research data infrastructure and clarifying the misconceptions on registry database, and discussing future development.
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.
ObjectiveWe constructed a real-world evidence evaluation system to provide reference for obtaining high-quality evidence in evidence-based medicine.MethodsThrough the investigation and analysis of the key factors influencing the real-world research evidence, combined with domestic and foreign literature and evaluation tools, we preliminarily constructed the indicators of the real-world evidence evaluation system, then consulted experts in related fields by the Delphi method, modified and determined the final evaluation indicators. ResultsThe indicators of the final real-world evidence evaluation system included 40 items. The recovery efficiencies of the two rounds of expert consultation were 88.2% and 100%; The expert coordination coefficients were 0.174 (P<0.001) and 0.189 (P<0.001). After the second round of consultation, the mean of Likert scale in the range of 3.73~4.93, and the coefficient of variation varied in the range of 0.05~0.21. ConclusionThe real-world evidence evaluation system constructed in this study has certain reliability and scientificity, which can provide a basis and help for the transformation of real-world research into high-quality evidence.
Assessing the clinical value of pharmaceuticals is crucial for comprehensive evaluation in clinical practice and plays a vital role in supporting decision-making for drug supply assurance. Real-world data (RWD) offers valuable insights into the actual diagnosis and treatment processes, serving as a significant data source for evaluating the clinical demand, effectiveness, and safety of drugs. This technical guidance aims to elucidate the scope of application of RWD for the clinical value assessment of pharmaceuticals, as well as the key considerations for conducting value assessment research. These considerations include identifying the dimensions of clinical value that necessitate RWD and effectively utilizing RWD for evaluation purposes. Additionally, this guidance provides essential points for implementing pharmaceutical clinical value assessment based on real-world data, with a specific focus on study design and statistical analysis. By doing so, this guidance assists researchers in accurately comprehending and standardizing the utilization of real-world research in conducting pharmaceutical clinical research.
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.