P60: Putting the “World” into Real World Evidence: The Evolving Global Landscape of RWE Regulatory Frameworks
Senior Director, Head EuCANZ Regulatory Policy
Pfizer Inc; EFPIA Ireland
The objective of this study is to analyze Real World Evidence (RWE) regulatory frameworks internationally (ex-US, ex-EU), to identify the barriers to fully embracing RWE for regulatory decision-making and to consider the impact on global drug development and registration.
During Q4 2020, an internal survey of Pfizer regulatory colleagues was conducted in 12 markets* to understand and benchmark the global regulatory policy landscape for RWE regulatory frameworks. *Australia, Brazil, Canada, China, India, Japan, Korea, Mexico, Russia, Singapore, Taiwan, Turkey.
Qualitative and quantitative data was gathered using twelve questions to assess:
1) The level of knowledge and interest by the regulatory authority on RWE
2) The status of RWE regulatory framework in each regulatory authority
3) Regulatory authority pilots and use cases
4) Type and degree of stakeholder engagement
5) Impact of the effective use of RWE on regulatory success
6) Rate limiting factors to effective use of RWE in regulatory decision-making.
The survey results demonstrate that regulatory authorities internationally are actively considering how to incorporate real-world data (RWD) and RWE into their regulatory frameworks and decision-making. Specifically, regulation, guidance or best practices on RWD and/or RWE are in place in Canada, China, Japan, Korea and Taiwan (including drafts under development). Moreover, several regulatory authorities have launched initiatives to increase their understanding and application of RWE in regulatory decision-making, notably in China, Korea, Singapore and Taiwan. Regulatory authorities are actively exploring the topic with outreach to stakeholders to gain insights and build knowledge (for example in Australia and Brazil).
Barriers to fully embracing the use of RWE in regulatory decision-making in the markets analyzed were identified and include the lack of a fully defined regulatory framework internationally (i.e. US FDA, EMA), lack of experience by the regulatory authority (including lack of case studies), regulator concerns about the quality of the data, data access, integration and analysis of electronic health records, and local legislation that requires clinical data.
Overall, the survey results indicate that the potential for RWD and RWE to impact regulatory success is most notable in relation to label expansion and as supportive data for initial registration, for example orphan/rare indications in oncology, rare disease or other small populations.
Regulatory authorities globally (i.e. beyond US FDA and EMA) are considering RWD beyond safety purposes with a rapidly increasing level of interest in RWE to support drug development. RWE is being increasingly leveraged globally to demonstrate treatment effectiveness, and to support and potentially expedite regulatory decisions. As a result, regulatory authorities globally are actively developing guidance on RWD for RWE, with many reaching out to industry to learn more about RWE strategies that companies are considering. The promise of RWE has been recognized for some time in the post-marketing setting. Now is the time to extend the role of RWE to the entire product lifecycle. To fulfil its true potential, all stakeholders must work together for RWE policies and approaches in the international markets that allow flexibility and further integration of RWE into the development, registration, and lifecycle process.