<已截止>2024/6/15 (星期六)【新藥開發系列】蛋白質新藥研發實務、法規解析與審查重點

1. 已核准上市的生物藥品與銷售 Sales and Approvals of Biologic Drugs

2. 蛋白質與小分子藥物CMC的差別 Differences in CMC of Protein and Small Molecule Drugs

3. 蛋白藥的製造 Manufacturing of Protein Therapeutics

• 技術與法規議題 Techniques and Regulatory Issues

4. 表現載體至細胞庫 Expression Vector to Cell Banking System

(1) 表現載體 Expression Vectors
(2) 細胞株挑選 Cell Line Selection
(3) 單細胞克隆和單克隆性 Single Cell Clone and Monoclonality
(4) 細胞庫的特徵與鑑定 Characterization of Cell Banks
(5) 細胞株的品質鑑定 Cell Line Qualification 
(6) 病毒檢測與鑑定分析 Viral Detection and Identification Assays

5. 原料藥/成品的製造與放行 Drug Substance/Product Manufacturing and Release

(1) 上下游製程 Upstream and Downstream Processing
(2) 外來病原之來源 Sources of Adventitious Agents
(3) 外來病原的管控與分析 Control and Analysis of Adventitious Agents
(4) 病毒清除研究 Viral Clearance Studies

• 縮小製程的效能 Validity of scaled-down process
• 具體注意事項 Specific precautions

(5) 原料藥特性與結構鑑定 Drug Substance Characterization
(6) 成品特性 Drug Product Characterization
(7) 管制和確效 Control and Validation
(8) 安定性試驗 Stability Studies

6. 蛋白質藥物之前臨床藥理與安全性研究 Preclinical Pharmacology and Safety Studies of Protein Drugs

(1) 蛋白質與小分子藥物非臨床性質的差異 Differences in Nonclinical Properties of Protein and Small Molecule Drugs
(2) 藥效學及安全藥理學 Pharmacodynamic Studies and Safety Pharmacology
(3) 基因毒性試驗 Genotoxicity Studies
(4) 單一劑量毒性試驗 Single Dose Toxicity Studies
(5) 重覆劑量毒性試驗 Repeated Dose Toxicity Studies
(6) 生殖與發育毒性試驗 Reproductive and Developmental Toxicity Studies
(7) 致癌性試驗 Carcinogenicity Studies

7. 蛋白質藥物之藥動學/毒動學/免疫原性研究 PK/TK/Immunogenicity Studies of Therapeutic Proteins

8. 研究用蛋白質產品的CMC審查和生產~主管機關的要求 CMC Review and Manufacturing in Investigational Protein Products ~Authorities Expect

(1) 臨床研究中的CMC和GMP目標 CMC and GMP Goals in Clinical Investigation
(2) 生物製品的主要考慮因素和研究用藥物的管控 Major Considerations of Biological Products and Control of Investigational Product
(3) 第一期臨床試驗的考量 Phase I Considerations

• CMC的內容及研究用藥物的管制 CMC Content and Control of Investigational Product
• 對藥物開發過程中分析方法的期望 Expectations For Analytical Methods During Development
• 分析方法確效 Analytical Method Validation
• 變更 Changes

(4) 臨床擱置問題 Clinical Hold Issues

9. 研究用蛋白質藥物之藥毒理審查~主管機關的要求 Pharm/Tox Review in Investigational Protein Products ~Authorities Expect

(1) FDA審查的非臨床研究類型 Types of Nonclinical Studies Reviewed by FDA
(2) 藥毒理專家審查的常見提問 Questions Asked by Review Pharmacologist/Toxicologist
(3) FDA對非臨床試驗的審查要求 What Does FDA Expect from Nonclinical Studies
(4) FDA對一般毒性試驗的審查要求 What Does FDA Expect in General Toxicology Studies

10. 生物相似性藥品 Biosimilars

(1) 生物相似性藥品的挑戰 Challenge of Biosimilars
(2) 生物相似性藥品的品質議題 Quality Issues of Biosimilars
(3) 生物相似性藥品的非臨床議題 Non-Clinical Issues of Biosimilars
(4) 生物相似性藥品的藥動學議題 Pharmacokinetics Issues of Biosimilars
(5) 生物相似性藥品的藥效學議題 Pharmacodynamic Issues of Biosimilars
(6) 生物相似性藥品的臨床議題 Clinical Issues of Biosimilars

11. 結論 Conclusion

12. 蛋白質藥物許可證申請 License of Protein Drugs

13. 其他資源 Some Further Resources