eal-world data (RWD) and real-world evidence (RWE) have played an increasingly important role in regulatory decision making in recent years. RWE has been commonly accepted by regulators for post-marketing safety surveillance activities but less so in the pre-marketing authorization phase. However, the pandemic provided an opportunity for regulators to assess the applicability of RWE to enable emergency use authorization or conditional approval of COVID-19 vaccines.

Australia

China

• January 2020: Guidelines for Real-World Evidence to Support Drug Development and Review (Interim) contained the definitions of real-world research, the status and scope of RWE, evaluation principles for RWE, and scientific principles on how industry can use RWE to support drug research and development.
• August 2020: Guidelines for Using RWE for Supporting Pediatric Drug Research & Development and Evaluations (Interim) (Chinese text only) provides guidance on how to use real-world research to support pediatric drug development.
• April 2021: Guidelines for RWD Used to Generate RWE (Interim) (Chinese text only) discusses RWD applicability and data curation, data security, and data quality.

NMPA, industry, and academia continue to engage; for example, a three-year NMPA RWE project aims to study worldwide RWE policies, collect use cases, and research data standardization. The upcoming annual NMPA RWE conference will further share research results and exchange of best practices among international professionals, and help the local regulatory landscape grow more multidisciplinary. These research results and use cases will also provide the scientific basis for new RWD/RWE regulatory guidelines in the future. These stakeholders are also discussing big data, digital health, personal information protection and cybersecurity, and other relevant areas that could influence future regulatory policy or guidance.

Japan

• June 2017: Basic principles on utilization of health information database for Post-Marketing Surveillance of Medical Products (Japanese text only)
• October 2017: Amendment of Ministerial Ordinance on Good Post-marketing Study Practice for Drugs (Japanese text only)
• February 2018: Points to Consider for Ensuring the Reliability of Post-marketing Database Study for Drugs
• December 2019: The Act on Securing Quality, Efficacy, and Safety of Products including Pharmaceuticals and Medical Devices (Japanese text only) was amended to clarify that academic societies, universities, institutions, and related organizations should cooperate in the appropriate use of pharmaceuticals and other medical products sold by marketing authorization holders, and collect information (including RWD) to ensure the appropriate use of pharmaceuticals and other medical products.
• March 2020: Points to Consider for Ensuring the Reliability of Post-marketing Database Study for Regenerative Medical Products
• July 2020: Basic principles in conducting a validation study of outcome definitions used for post-marketing database study (Japanese text only)
• March 2021: Basic Principles on Utilization of Registry for Applications and Points to Consider for Ensuring the Reliability in Utilization of Registry Data for Applications address registry data utilized during a product’s lifecycle from pre-approval, re-examination, application after conditional and time-limited approval, to labeling update.

In April 2021, the PMDA established its RWD working group to discuss regulatory issues related to RWD/RWE, such as data reliability standards and methodological approaches, and seeks to continuously promote utilization of RWD in the Japanese regulatory framework. PMDA published a summary of results of studies utilizing the medical information database network MID-NET, which evaluates the safety of drugs based on electronic medical information accumulated and registered by daily medical practice in medical institutions.

South Korea

The Ministry of Food and Drug Safety (MFDS) has displayed keen interest in leveraging RWE for regulatory decision making, even with no formal RWE regulatory framework established, and has developed a comprehensive roadmap by sequentially expanding the scope and implementation of RWE utilization:

• December 2019: MFDS published the Guideline on Risk Management Plan for Medicines to allow post-marketing safety study based on database study using RWD (Korean text only)
• June 2021: MFDS published the Guideline for Medical Information Database Research (Korean text only) on utilizing RWE that analyzed RWD, such as national health insurance data and electronic medical records, for post-marketing safety studies
• July 2021: MFDS conducted the virtual symposium Global trends and implications of using RWE to establish a drug safety management system
• December 2021: MFDS organized the Using Big Data for Regulatory Science Decision-Making regulatory forum.

The Korea Comprehensive Plan for Drug Safety Management (2020-2024) anticipates that MFDS will continue this momentum by building a local ecosystem to leverage innovative technology including RWE to support new drug development.

Taiwan

RWE has become increasingly relevant in regulatory decision-making in Taiwan, with the Taiwan Food and Drug Administration (Taiwan FDA) issuing numerous guidelines (all Chinese text only):

• July 2020: Basic Considerations for Real-World Evidence to Support Drug Research and Development
• January 2021: RWE Study Designs – Pragmatic Clinical Trial Considerations
• March 2021: RWD – Assessment and Consideration of Relevance and Reliability
• July 2021: Precautions for Using RWD/RWE as Technical Documents for Applying for Drug Review

Based on the comprehensive scope of these guidance documents, the Taiwan FDA appears to have high interest in improving local procedural and methodological practices to advance regulatory-quality RWE in Taiwan.

Conclusion

• In China, NMPA’s RWE guidance discusses the use, evaluation, and scope of RWE in China during drug development and lists several scenarios in which RWE may be used to support drug development and regulatory decisions. NMPA also released guidance specific to the use of RWE to support pediatric drug development.
• In Japan, PMDA’s RWE guidance focuses on leveraging post-marketing studies and registry data for product approval applications.
• Taiwan FDA’s RWE guidance discusses how RWE can complement traditional randomized clinical trials for rare diseases or new indications.
• Data reliability and ensuring that data is of high quality and fit for purpose is a common theme among these three agencies.

There have been multiple efforts across Asia Pacific to enhance the use of RWE for regulatory decision making and RWE research is under discussion in other Asian-Pacific markets, with several workshops or research activities presented in Singapore, Malaysia, Hong Kong, India, and Thailand. The region is expected to collaborate with other international agencies to ensure a more coordinated RWE environment around the world.