In 2019, the national government issued the document "Implementation Plan for Supporting the Construction of the Boao Lecheng International Medical Tourism Pilot Area", which allowed the use of innovative drugs and medical devices in medical institution of Boao Lecheng. These medical products had been designed to meet urgent clinical requirements and had been approved by regulatory authorities overseas. Through the use of these medical products, real-world data were generated in the routine clinical practice, based on which real-world evidence might be produced for regulatory decision-making by using scientific and rigorous methods. In March 2020, the first medical device product using domestic real-world data was approved, suggesting that the real-world data initiative in Boao Lecheng achieved initial success. This work also provided important experience for promoting the practice of medical device regulatory decision-making based on real-world evidence in China. Here, we shared the preliminary experiences from the study on the first approved medical device product and discussed the issues on developing a real-world data research framework in Boao Lecheng in attempt to offer insights for future studies.
Evidence-based medicine is the methodology of modern clinical research and plays an important role in guiding clinical practice. It has become an integral part of medical education. In the digital age, evidence-based medicine has evolved to incorporate innovative research models that utilize multimodal clinical big data and artificial intelligence methods. These advancements aim to address the challenges posed by diverse research questions, data methods, and evidence sources. However, the current teaching content in medical schools often fails to keep pace with the rapidly evolving disciplines, impeding students' comprehensive understanding of the discipline's knowledge system, cutting-edge theories, and development directions. In this regard, this article takes the opportunity of graduate curriculum reform to incorporate real-world data research, artificial intelligence, and bioinformatics into the existing evidence-based medicine curriculum, and explores the reform of evidence-based medicine teaching in the information age. The aim is to enable students to truly understand the role and value of evidence-based medicine in the development of medicine, while possessing a solid theoretical foundation, a broad international perspective, and a keen research sense, in order to cultivate talents for the development of the evidence-based medicine discipline.
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.
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.
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.
As an important source for real-world data, existing health and medical data have gained wide attentions recently. As the first part of the serial technical guidance for real-world data and studies, this report introduced the concepts, features and potential applications of existing medical and health data, proposed recommendations for planning and developing a research database using existing health and medical data, and developed essential indicators for assessing the quality of such research databases. The technical guidance may standardize and improve the development of research database using existing health and medical data in China.
Real-world data is been increasingly valued nowadays. This paper combined with related requirements of clinical evaluation of medical devices in China, studied the role of real-world evidence in pre-marketing clinical evaluation of medical devices in terms of technical evaluation, in aim of providing reference for the future application of China's real-world evidence in pre-marketing clinical evaluation.
Given the growing importance of real-world data (RWD) in drug development, efficacy evaluation, and regulatory decision-making, establishing a scientific and systematic data quality regulatory framework has become a strategic priority for global pharmaceutical regulatory authorities. This paper analyzed the EU's advanced practices in RWD quality regulation, compared the RWD quality regulatory systems of China and the EU, and aimed to derive implications for enhancing China's own framework. The EU has made significant progress by promoting the interconnection, intercommunication, and efficient utilization of data resources, implementing a collaborative responsibility mechanism spanning the entire data lifecycle, developing a standardized, tool-based quality assessment system, and facilitating international cooperation and alignment of rules. While China has established an initial regulatory system for RWD quality, it still confronts challenges such as unclear mechanisms for data acquisition and utilization, underdeveloped operational standards, and unclear responsibility delineation. In contrast, by adapting relevant EU experience, China can refine its regulatory framework, establish mechanisms for the interconnection, intercommunication, and efficient utilization of RWD, develop more practical quality assessment toolkits, improve the lifecycle responsibility-sharing mechanism, and promote the alignment of RWD quality regulation with international standards. These enhancements will advance the standardization and refinement of RWD quality regulation in China, ultimately strengthening the scientific rigor and reliability of regulatory decisions.
A patient registry database is an important source of real-world data, and has been widely used in the assessment of drug and medical devices, as well as disease management. As the second part of the serial technical guidance for real-world data and studies, this paper introduces the concept and scope of potential uses of patient registry databases, proposes recommendations for planning and developing a patient registry database, and compares existing health and medical databases. This paper further develops essential quality indicators for developing a patient registry database, in expect to guide future studies.
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.