生物新药开发与市场课件.pptVIP

永昕生物醫藥 生技產業導論 生物新藥開發與市場 生物製劑的種類 Therapeutics Gene Therapy In Vivo Diagnostics Therapeutic Vaccine BioDevice Tissue Repair Xenotransplantation Natural Product 生物製劑的特性 複雜高分子物質 三度空間結構特性 生物有效性或活性 需特殊安全測試 Critical Parameters for Biologics Safety (安全性） Potency（強度） Purity （純度） Consistency （持續性） Efficacy （有效性） 新藥開發、製造、測試及申請流程 Phases and Quality Pre-Clinical - Close to cGMP Phase I - cGMP w/ some changes Phase II - cGMP w/ changes Phase III - cGMP w/ No Changes BLA - No More Product Development Process Development - Mission To develop production processes of appropriate scale - from pre-clinical exploration to commercialization Process Validation Cell Culture Filtration Chromatography Viral Clearance Aseptic Manipulation Validation Equipment Other - cleaning, storage,transferring, packaging, and shipping Manufacturing Scheme Manufacturing - Outline Master Cell Bank Working Cell Bank Starting Material Production Purification Modification Formulation Aseptic Vialing Labeling Clinical Trials Pre-Clinical IND Animal studies, safety, metabolic, pharmacological process description Phase I: Human Safety Clinical pharmacology studies Clinical Trials Phase II: Efficacy Controlled studies in patients with disease or condition Blinded clinical evaluation Clinical Trials Phase III: Safety and Efficacy Both controlled and open trials Closely monitored by sponsor for adverse reactions and information to be used for setting dosage requirements and labeling. Clinical Trials Treatment: (Physicians IND; Compassion) Involves patients with a serious, life threatening, or terminal illness when alternative treatment is not available or not effective and proposed treatment use has some evidence of effectiveness. Documentation Control To reduce the risk of error due to oral or informal communication, to provide clear, written instructions, and to permit traceability. Document Training Record Specifications (supplies, chemicals, products) SOPs Production Batch Records Validation Records