Archives: Agenda

• Regulatory Compliance: Navigating strict NMPA oversight and IP-GMP rules for MRCT integration.
• Sourcing Strategy: Utilizing local procurement and bonded zones to bypass customs delays.
• Labeling Standards: Implementing mandatory Simplified Chinese text while maintaining blinding integrity.
• Domestic Logistics: Managing secure cold-chain distribution networks across vast hospital networks.
• Chain of Custody: Executing meticulous drug reconciliation leading to certified local destruction.

• 法规合规：应对 NMPA 严格监管以及 MRCT 整合中的 IP-GMP 规则。
• 采购策略：利用本地采购和保税区政策避免清关延误。
• 标签标准：落实强制性简体中文文本要求，同时保持盲法完整性。
• 国内物流：在广泛的医院网络中管理安全的冷链配送网络。
• 监管链：执行细致的药品核对，直至获得认证的本地销毁

As more Chinese biotech companies expand clinical development beyond China, the question is no longer whether to go global, but how to build globally ready development programs from the outset. Drawing on practical experience and industry observations, this presentation will explore the critical decisions sponsors face across development planning, regulatory strategy, study execution, and operational readiness. It will also share common challenges, lessons learned, and practical considerations to help organizations better prepare for clinical development across the US, Europe, and other international markets.

随着越来越多中国创新药企业加速国际化布局，当前面临的关键问题已不再是是否走向全球，而是如何从研发早期建立符合全球要求的临床开发策略。本次分享将结合实践经验与行业观察，探讨企业在全球临床开发过程中面临的关键决策，包括开发规划、注册策略、研究执行及运营准备等内容，并分享常见挑战、实践经验和可借鉴的建议，帮助企业更好地规划美国、欧洲及其他国际市场的临床开发。

• Leveraging China’s integrated research, healthcare, and technology networks to accelerate development
• How scale, speed, and digital innovation are reshaping global clinical trial execution
• Translating local operational excellence into global study success
• Less on drug supply multinational sponsors can apply from China’s evolving clinical ecosystem

• 借助中国一体化的科研、医疗和技术网络加速开发。
• 规模、速度与数字化创新如何重塑全球临床试验执行。
• 将本土运营卓越转化为全球研究成功。
• 跨国申办方可从中国不断演进的临床生态中借鉴的经验。

This session outlines an end-to-end framework for Multi-Regional Clinical Trials (MRCTs). It covers critical regional epidemiology and regulatory planning, third-party operational models, the impact of inter-ethnicity differences on future labeling, and navigating health agency registration decisions.

Plan

• Clinical epidemiology in different regions
• Regulatory requirements on protocol design and patient recruitment
• Timeline and financial models

Do

• Operational models involving third parties

Check

• Influence of inter-ethnicity differences and response differences on future label statements

Access

• Agency‘s decision on registration

本演讲将全面概述多区域临床试验（MRCT）的端到端框架：涵盖区域流行病学与法规落地规划（Plan）、第三方运营模型构建（Do）、种族差异对未来说明书标签的影响评估（Check），以及应对监管机构注册决策的策略（Access）。

• The 3+3 design is simple but has limited statistical performance, failing to meet modern Phase I R&D needs.
• BOIN is a model-assisted design whose core centers on constructing optimal decision intervals around the target toxicity rate.
• BOIN combines strong statistical performance with high clinical feasibility.
• Compared to 3+3, BOIN allocates patients more rationally and improves MTD accuracy.
• BOIN has evolved into a design family that extends to efficacy, combination therapies, late-onset toxicities, and OBD optimization.

• 3+3 简单但统计性能有限，已难满足现代 I 期研发需求。
• BOIN 是模型辅助设计，核心是围绕目标毒性率构建最优决策区间。
• BOIN 兼具较强统计性能与较高临床可实施性。
• 相比 3+3，BOIN 能更合理分配患者并提高 MTD 准确率。
• BOIN 已发展为设计家族，可扩展到疗效、联合用药、迟发毒性和 OBD 优化等场景。

• How to build a “Predict and Prevent” Supply Mindset
• Dynamic Re-supply Models for a Dynamic Clinical Reality
• A Step-by-Step Guide to Smoother Cross-Border Movements
• Rapid Response Protocols and Effective Contingency Planning
• Integrating Partners into a Single, Seamless Team

• 如何建立“预测并预防”的供应思维。
• 面向动态临床现实的动态补货模型。
• 更顺畅跨境运输的分步指南。
• 快速响应流程与有效的应急规划。
• 将合作伙伴整合为一个无缝协作团队。

Moderator: Jae-Young Ha, CBO, AriBio

• Proof of concept established: 2.5-year follow-up data for OTOF gene therapy demonstrate that inner-ear gene therapy is safe, effective, and delivers predictable therapeutic outcomes.
• The target-to-IND pathway is mature: parallel workflows + a dense CRO/CDMO ecosystem + an integrated CRDMO model enable China to move from discovery to IND 50–70% faster than the global norm.
• Supply chain readiness determines successful delivery: cold-chain logistics, potency testing, and end-to-end regulatory compliance systems (NMPA/QMS) are critical to the scaled delivery of cell and gene therapies (CGT).
• Structural advantage: national strategic tailwinds + partners’ end-to-end delivery capabilities help Chinese companies such as Euhearing Therapeutics accelerate CGT implementation.

• 概念验证已成立: OTOF 基因治疗 2.5 年随访数据证明内耳基因治疗安全、有效且疗效可预测。
• 从靶点到 IND 已成熟: 并行化工作流 + 密集 CRO/CDMO 生态 + CRDMO 一体化,使中国「发现→IND」周期比全球快 50–70%。
• 供应链就绪决定落地: 细胞与基因治疗(CGT)的冷链、效价与全程监管链(NMPA/QMS)是规模化交付的关键。
• 结构性优势:国家战略红利 + 合作伙伴端到端交付，有助于以佑音医药等为代表的中国企业加速 CGT 落地。

Forcing a single clinical supply model across APAC creates delays, costs, and regulatory risks. This presentation addresses regional complexities through flexible strategies and local collaboration.

• Model Selection: Evaluating central, regional, and hybrid supply frameworks.
• Streamlined Logistics: Bridging gaps between sourcing, compliance, and local distribution.
• Risk Mitigation: Overcoming cross-border bottlenecks and critical documentation challenges.
• Strategic Value: Balancing speed, cost, and long-term partner alignment over transactional relationships.

在亚太地区强推单一的临床供应链模型会导致延误、高昂成本和合规风险。本演讲将通过灵活的战略与本地化合作，探讨并解决这些区域复杂性问题。

• 模式选择：评估集中式、区域式以及混合式的供应链架构。
• 高效物流：消除采购、合规与本地分销之间的断层。
• 风险缓解：克服跨境瓶颈与关键文件审查带来的挑战。
• 战略价值：权衡速度与成本，建立长期的伙伴协作，而非单纯的业务交易