PURPOSE: To discuss the developments of using real-world evidence (RWE) to support regulatory evaluation on medical devices in US and China.

DESCRIPTION: Randomized controlled trials (RCT) has been considered gold standard to demonstrate the safety and efficacy of a medical device. However, practical limitations require alternative approaches to RCT and increased flexibility in trial design and statistical analysis. There are new developments in US (RWE guidelines) and China (free trade pilot zone) supporting the regulatory decisions for devices.

Dr. Du will discuss common issues in regulatory submissions using RWE in US. Quality of RWE varies by device type, sponsor experience and RWD sources. FDA assesses RWD relevance and reliability to determine whether RWE derived from particular RWD is qualified. Sponsor should clearly demonstrate its RWE fits for the regulatory purpose with acceptable quality.

Dr. Shi will analyze the RWE test cases by the National Evaluation System for health Technology (NEST) and provide insights about how to efficiently consolidate RWE from clinical registries, EHRs, claims, and other sources to inform device development and evaluation, and to support regulatory decision-making throughout lifecycle.

In China, the policy environment is evolving towards the use of RWE to support regulatory decision-making for devices. Recently, a unique opportunity has been introduced for devices to gain faster access by leveraging RWE generated in a free trade pilot zone in Bo’ao Lecheng, Hainan Province.

A successful Bo’ao case will be shared where safety and effectiveness of XEN^® in Chinese patients with refractory glaucoma were collected and RWE were generated to enable the assessment of ethnic differences to treatment with XEN® and gain regulatory approval. With its continuing development, we anticipate more successful approvals of innovative devices and drugs.

The workshop will benefit the audience to understand new developments, nuances, opportunities, and challenges in using RWE with regulatory pathways.