细胞与基因治疗讨论组（CGT DG）：战略路线图交付进展.pdf

1、International Council for Harmonisation of Technical Requirementsfor Pharmaceuticals for Human UseCell&Gene Therapy Discussion Group(CGT DG):Progress toward Delivering a Strategic RoadmapCASSS CMC Strategy Forum JapanKathleen Francissen,Ph.D.BIO10 December 20242OverviewDescribe the Cell&Gene Therapy

2、 Discussion Group(ICH CGT DG)Provide rationale for CGT DG formationOutline the remitDescribe progress toward deliverables in the Work Plan3Cell&Gene Therapy Discussion GroupTopic adoption date:August,2023Rapporteur:Dr.Kathleen Francissen,BIO Regulatory Chair:Dr.Melanie Eacho,FDA,United StatesCGT DG

3、Kick-off Meeting:October,2023Work Plan endorsement:December,2023Rapporteur Supporter:Elaine Shults,BIOICH Website4Cell&Gene Therapy Discussion GroupCGT DG Remit PaperCGT DG Work PlanCGT DG Roster5Remit PaperRemit paper describes scope and timelinesFinal deliverable by October,2025625 member organiza

4、tionsrepresenting 45 countriesICH CGT DG Members7ICH CGT DG MembersRegulatory/Administrative AuthoritiesEU commission,EuropeFDA,USAMHLW/PMDA,JapanANVISA,BrazilEDA,EgyptHSA,SingaporeMFDS,Republic of KoreaMHRA,UKNMPA,ChinaSFDA,Saudi ArabiaTFDA,Chinese TaipeiHealth Canada,CanadaSwissmedic,SwitzerlandAN

5、MAT,ArgentinaANPP,AlgeriaIndustry AssociationsEFPIAJPMAPhRMABIOIFPMAIGBAOther/International AssociationsEDQMUSPIPRPWHO8Cell&Gene Therapy Discussion GroupTechnical discussion forum Ultimate deliverable:Strategic Roadmap9Rationale for forming CGT DGNumerous studies:estimate 1400 active ATMP clinical t

6、rials worldwide.*Largest numbers are:-CAR T-cell products and-Adeno-associated viral(AAV)vector-based gene therapy productsEstimate 5,800 patients dosed with AAV-based GT and 38,000 with CAR T-cell products(including clinical+commercial setting).This figure refers to Advanced Therapy Medicinal Produ