P319: Bridging Study Evaluation and Regulatory Pathways for New Drug Applications: Addressing Ethnic Factors in Taiwan
Poster Presenter
Jun-Fon Wang
Senior Clinical Reviewer
Center For Drug Evaluation (CDE), Taiwan Taiwan
Objectives
Following the release of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) E17 in 2017, multi-regional clinical trials (MRCTs) became the primary approach to drug development. In Taiwan, bridging study evaluation (BSE), based on ICH E5(R1), assesses whether global data can be extrapolated to East Asian populations and influence New Drug Applications (NDA) outcomes.
Method
This study included all BSE cases submitted to the TFDA and reviewed by the CDE between 2018 and 2024. Under current regulations, biosimilars and drugs for pediatric or rare severe diseases are exempt. Assessment reports and prescribing information were retrieved from TFDA and CDE databases.
Results
During the study period, 245 cases were submitted for BSE: 89.80% new chemical entities, 5.30% new combinations, 2.45% new dosage forms, 1.63% new routes of administration, and 0.82% biosimilars. These involved 223 drug substances: 59.18% small molecules, 36.74% biologics (including vaccines, peptides, and protein therapeutics), 3.67% antibody–drug conjugates, and 0.41% gene therapies. Therapeutic classes =5% were antineoplastic and immunomodulating agents (36.77%), anti-infectives for systemic use (19.73%), and nervous system agents (11.21%).
Among 126 cases granted a full BSE waiver, 88.10% obtained NDA approval; among the 32 cases without a waiver, 6.25% obtained NDA approval (2 successful NDA cases: one conducted a confirmatory trial in Taiwan, and the other was exempted from BSE due to designation as a rare and severe disease) while 78.12% did not proceed to NDA submission.
Of the 87 cases in which ethnic-specific regulatory considerations were stated in the BSE waiver letter. These considerations, as documented in the waiver letters, involved East Asian efficacy and safety data (39.08%), efficacy (19.54%), safety (16.09%), Pharmacokinetics (PK) and Pharmacodynamics (PD) (9.20%), combined efficacy, safety, and PK/PD (6.90%), efficacy and PK/PD (5.75%), or safety and PK/PD (3.44%).
Of the 87 cases with ethnic-specific considerations documented in BSE waiver letters, 85.06% (74 cases) received NDA approval; two were consolidated into a single submission, resulting 73 NDA approvals. Among approved cases, East Asian-specific concerns persisted in efficacy (50.68%), safety (36.99%), PK/PD (17.81%), and genomics (2.74%).
To address these concerns, TFDA and CDE adopted post-marketing requirements (27.34%), labeling modifications (21.09%), post-marketing studies conducted in or including Taiwan (17.17%), risk management plans (12.50%), enhanced pharmacovigilance (4.69%), and indication restrictions (3.91%); 13.28% required no specific action.
Conclusion
The findings illustrate the evolving role of BSE within the NDA pathway under the ICH E5(R1) and ICH E17 framework. By incorporating ethnic-specific evidence into regulatory decision-making, BSE supports structured risk management and tailored regulatory actions while maintaining alignment with global harmonization and facilitating patient access.
From a regulatory perspective, these findings also reflect the practical considerations of the TFDA in evaluating the adequacy of bridging evidence and determining appropriate regulatory decisions and measures, including post-marketing requirements (e.g., studies conducted in or including Taiwan), labeling modifications, risk management plans, enhanced pharmacovigilance and indication restrictions.
Collectively, these findings suggest that early and proactive dialogue between marketing authorization holders (MAHs) and Taiwanese regulatory authorities during drug development may further improve regulatory efficiency. In particular, early engagement—such as incorporating Taiwan into the design of multi-regional clinical trials (MRCTs)—may help streamline regulatory review and expedite market authorization in Taiwan.
