生物新药开发与市场ppt课件

1、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q複雜高分子物質複雜高分子物質q三度空間結構特性三度空間結構特性q生物有效性或活性生物有效性或活性q需特殊平安測試需特殊平安測試Critical Parameters for Biologics Safety (平安性平安性 Potency強度強度 Purity 純度純度 Consistency 持續

2、性持續性 Efficacy 有效性有效性新藥開發、製造、測試及申請流程新藥開發新藥開發Drug DevelopmentDrug Development臨床前動物試驗臨床前動物試驗Pre-clinicalPre-clinicalDevelopmentDevelopment申請審核新藥物申請審核新藥物Investigational New DrugInvestigational New DrugApplication (IND)Application (IND)三階段人體臨床試驗三階段人體臨床試驗Clinical DevelopmentClinical Development申請藥物許可申請藥物許

3、可BLABLA上市上市Introduction/DistributionIntroduction/DistributionTo the MarketTo the MarketPhases 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 DevelopmentProcess Development - MissionTo develop pro

4、duction processes of appropriate scale - from pre-clinical exploration to commercializationProcess Validation Cell Culture Filtration Chromatography Viral Clearance Aseptic Manipulation Validation Equipment Other - cleaning, storage,transferring, packaging, and shippingManufacturing SchemeManufactur

5、ing - Outline Master Cell Bank & Working Cell Bank Starting Material Production Purification Modification Formulation Aseptic Vialing LabelingClinical Trials Pre-Clinical & INDAnimal studies, safety, metabolic, pharmacological & process description Phase I: Human Safety Clinical pharmaco

6、logy studiesClinical Trials Phase II: Efficacy Controlled studies in patients with disease or condition Blinded clinical evaluationClinical 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

7、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 Co

8、ntrolTo 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 + Equipment IQ/OQ/PQ Equipment use logCO

9、NTROLA documented process by which validated procedures are consistently followed to eliminated unacceptable deviations from the established standards.CONTROL General Controls Design Control Facility Control Equipment Control Material/Component Control Operational Control Finished Product Control生物新藥市場評估需求評估需求評估New Case / YearTotal Case / Year用藥量用藥量市場占有率市場占有率市場治療費用年市場治療費用年生物新藥市場評估 製作評估Patent /法律問題製造困難度量產保管體內外活性劑量單一重