1.1 Utilization of Real-World Data (RWD) in Healthcare

Real-world data (RWD) refers to patient health status and healthcare delivery information collected from various sources outside traditional clinical trials. The analysis of RWD generates real-world evidence (RWE) and offers clinical insights into a medicinal product’s potential benefits or risks [1].

In recent years, with incentives for developing comparative effectiveness research evidence to improve the care delivery, the use of RWD has been growing. The rapid generation of various types of digital data has provided valuable and diversified RWD sources [2]. Current RWD sources contain healthcare data, patient and/or disease registries, claims data, environmental data, and health-related data generated from mobile devices, social media, and patient platforms [3]. Through different study designs (e.g., retrospective/prospective cohort, case-control, or pragmatic clinical trial), these RWD sources have transformed values from post-marketing safety evaluation to broader applications in healthcare. RWE generated from RWD can support identifying unmet medical needs, optimizing drug development strategy, and assessing drug safety and effectiveness among diverse populations in the real-world setting [3]. Beyond therapeutic development, RWE can also provide insights into informing clinical guidelines and changing clinical practice, which are essential for patient experience optimization for better clinical outcomes [4].

Enhanced data quality promotes the utilization of RWD. Digitalization has been extensively integrated into clinical practice. The accumulation of digitalized data contributes to the generation and linkage of large electronic datasets containing comprehensive data elements in the real-world clinical setting. Currently, many RWD sources have the potential to ensure the performance of statistical and machine learning procedures and result reproducibility and replicability, leading to valid RWE generation with savings in both cost and time and enhancing the efficiency of medical and health-related research and decision making [2]. Enhanced RWD quality has also facilitated global collaboration by establishing a catalogue of RWD sources and harmonizing these data for cross-country collaborative research [5].

Real-world study (RWS) design and methodology have also been advanced, which facilitates the utilization of RWD. Based on advances in data quality and linkage, registry-based randomized control trials (RCTs), which are defined as pragmatic trials that use registry data for case records, data collection, randomization, and follow-up, are playing an important role in answering clinical questions pragmatically and cost effectively [6, 7]. One example is the national-level SWEDEHEART (Swedish Web-system for Enhancement and Development of Evidence-based care in Heart disease Evaluated According to Recommended Therapies) registry database [8]. The advanced statistical techniques and structured process have also allowed the emulation of RCTs with high-quality RWD to agree on the conclusions [9]. With data suitable for study questions, design and analysis, the causal effects of therapies can be evaluated through both RCTs and observational RWSs. Therefore, RWD may offer causal insights when trial data are either unavailable or cannot be generated quickly or feasibly, which has enhanced the credibility of RWD in research [10].

RWD utilization has obtained regulatory recognition. In 2016, the United States Food and Drug Administration (US FDA) took a significant step by introducing the 21st Century Cures Act, aiming to assess the potential use of RWE in regulatory decision-making processes, including fulfilling post-approval study requirements, extending drug labels, and considering RWE as an external comparator for new drug submissions [11]. Following this milestone, the National Medical Products Administration (NMPA), the European Medicines Agency (EMA) and the Japan's Pharmaceuticals and Medical Devices Agency (PMDA) subsequently issued guidance on RWE application in drug development and addressing the methodological and operational aspects concerning the use of RWE [12,13,14]. These collective efforts reflect the growing acceptance of RWE as trustworthy information about the safety and effectiveness of drug therapies.

1.2 Importance of RWD/Real-World Evidence (RWE) in China’s Healthcare Context

In China, the release of “Healthy China 2030” has played a significant role in driving digitalization in healthcare facilities, to enhance the accessibility of medical big data nationwide [15]. The adoption of digital healthcare systems (e.g., Hospital Information System) across hospitals has offered a rich source of RWD. These RWD sources are being utilized to support robust analyses and enhance the generalizability of research findings in real-world clinical settings among the broad Chinese population [16].

RWE is gaining wide attention in China, and the number of published, peer-reviewed articles reporting RWS has continued to increase [17]. In China, RWE arose to compensate for the limitations of traditional clinical trials and provides additional evidence to inform clinical practice and policy decisions [17]. RWD/RWE gained regulatory recognition from 2019 onwards following the issue of relevant guidelines by NMPA [18]. The value of RWD/RWE is fostering collaboration and partnerships across healthcare stakeholders and initiating public–private partnership models or muti-stakeholder alliances to some extent in China.

1.3 External Advisory Committee (EAC) Meetings

A multi-stakeholder External Advisory Committee (EAC) was established to promote collaboration and partnership among diverse healthcare stakeholders and leverage the impact of China RWE globally [19]. Meetings were initiated among the EAC members. These meetings gathered experiences and insights from diverse healthcare stakeholders intending to enhance patient experience by applying RWD and RWE, particularly in the context of China. See the electronic supplementary material (ESM, including Table A1 and Table A2) for a detailed introduction on the EAC and its members.

Three EAC meetings with different focus topics were held quarterly in 2023 to collect insights and suggestions. In total, nine experts from China, one from South Korea, and two from Europe (Sweden and the Netherlands) were selected as EAC members and attended the series of meetings. In each meeting, Chinese experts shared their cases and thoughts encompassing the current and evolving RWD landscape and RWE enablement in driving clinical practice change in China, followed by presentations from international experts to introduce RWD development in other countries, and/or multistakeholder partnership and collaboration at the global level. Each presenter referred to facts in the literature/guidelines or their research published in peer-reviewed articles. Points presented in these meetings led to panel discussions. Panel discussion was conducted to identify challenges and opportunities for RWD quality enhancement and strategic partnership development to unlock RWD potential in China.

Experts’ presentations grouped by theme were summarized, including the RWD landscape in China, RWE enablement in China, and the patient-centric ecosystem to maximize the RWE/RWD value at a global level. Figure 1 shows an overview of the objectives of the three EAC meetings.

Fig. 1

An overview of the objectives of three External Advisory Committee meetings. EAC External Advisory Committee, RWD real-world data, RWE real-world evidence

Based on the insights and experiences obtained from the EAC meetings, we aim to:

1.

Understand the current and evolving RWD landscape in China.

2.

Articulate the values of RWE in ensuring Chinese patients’ equitable access to affordable medicines and solutions.

3.

Identify strategic opportunities and partnerships for expansion of RWE generation in China.