Session (CTI)

ISSUE: China’s biopharmaceutical ecosystem is moving from “in China for China” to a bidirectional evidence model linking China and ex-China decisions. China’s stronger science, faster R&D, and growing pipeline are driving multinationals to source Phase 1/2 assets from Chinese innovators, increasing demand for China-generated clinical data to support global development (“in China for ex-China”). When trial design, execution, and quality align with global standards, these data are increasingly portable, enabling earlier development, indication expansion, and post-approval commitments. China real-world evidence (RWE), however, is not yet broadly transferable for ex-China payer/HTA use. Gaps in data provenance, coding, care pathways, representativeness, and governance limit comparability. This clinical-versus-RWE split raises practical questions about what China evidence can support globally today, and what must change for RWE to contribute tomorrow. Rare disease is a key proving ground. Diagnostic uncertainty, thin epidemiology, and heterogeneous care are major access and value barriers worldwide. In China, uneven diagnosis and limited natural-history data can deepen uncertainty; yet lower-cost, high-speed study infrastructure may enable faster trials and natural-history cohorts that strengthen both China filings and ex-China indication expansion. The landscape demands a new evidence playbook. OVERVIEW: Presentations (~15 minutes each) will cover: • Why China clinical evidence is becoming globally acceptable and how to make trials exportable. • Why China RWE remains limited for ex-China use and what advances could narrow the gap. • Bidirectional frameworks for using ex-China evidence in China and China evidence ex-China, including hybrid designs. • Rare disease methods to reduce diagnostic/epidemiologic uncertainty and enable indication expansion. The panel will conclude with audience debate to identify principles and guardrails for integrating China and global evidence streams to improve patient access.