The Brazilian regulatory system for cell therapy products

Transcription

1 The Brazilian regulatory system for cell therapy products Brasília 23 de outubro de 2018 Gerência de Sangue, Tecidos, Células e Órgãos GSTCO Diretoria de Autorização e Registros Sanitários - Diare Agência Nacional de Vigilância Sanitária - Anvisa

2 BLOOD, TISSUES, CELLS, ORGANS PRODUCTS Conventional Therapy minimally manipulated cells, tissues, organs and intended for homologous use only. Not involve the combination. Tissues and cells for reproductive use Blood e components for transfusion Hematopoietic progenitor cells for bone marrow transplantation Tissues and organs for transplants - Establishment Registration - Periodic inspection based on risks - GMP in Cells, Blood and Tissues - Haemovigilance/Biovigilance risk-based approach Advanced therapy substantially manipulation cells or tissues or innovation in original function (not intended to be used for the same essential function) Somatic cell therapy products Tissues engineered products Gene therapy products (in vivo and ex vivo) ADVANCED THERAPY PRODUCTS (ATMP)

3 Project Brazilian Regulatory Framework for ATMP Cell Products Biologics Products Advanced Cell Therapy Somatic cell products therapeutics recombinant genes products (gene therapy in vivo) Gene Therapy ex vivo Tissue engineered product Cell products wich recombinant nucleic acid Cell engineered in tissues products Good Cell Practice Resolution n 214/2018 Gene Therapy ex vivo

4 Project Brazilian Regulatory Framework for ATMP ATMP CLASS II Substantially manipulation Combined productos; Gene Therapy Products Minimally manipulated wich innovation in original function ATMP CLASS I RISKS APPROACH

5 2005 Biosafety Law use of embrionic stem cells in Brazil 2012 Regulatory Agenda Clinical Trials with ATMP 2015 Regulatory Agenda- Marketing Authorization with ATMP 2018 RDC 214/18 GMP on ATMP 2004 Progenitor Cells for Bone Marrow Transplant 2010 New Regulation for Progenitor Cells for Bone Marrow Transplant 2011 RDC 09/11 Center of Technology Cells (CTC) 2016 Decision to aprove commercialization of ATMP 2017 Public consultation Clinical Trial ATMP

6 Classification sheet Roteiro/Guia de Inspeção 2018 RDC XX clinical trial with ATMP 2019 RDC XXX Marketing Authorization on ATMP Guidance documents 2018 CP XX Registros de Produtos de Terapias Avançadas 2019 RCD XX GMP certification on ATMP Biofarmacovigilance

7 Project Brazilian Regulatory Framework for ATMP Regulatory Classification as ATMP (early development) Anvisa wich suport by CAT ATMP CLASS I Clinical Trials application (Simplified) ATMP CLASS II Clinical Trials application (Complete) Main concerns - Safety and Efficacy Automatic start studies with submission application Approval in Ethics Committee (CONEP) Monitoring by risk-based inspection program Main concerns Safety, Quality and Efficacy Approval of Anvisa to start studies Approval in Ethics Committee (CONEP) and Biosafety Commission (CTNBio) Monitoring by risk-based inspection program

8 Project Brazilian Regulatory Framework for ATMP Development No-clinical Clinical Trials conditional marketing authorization Marketing Authorization Simplified Marketing Authorization Good Manufacturing Practices Biovigilance approved clinical trials Presented by Patrick Celis on 29 May 2018

9 Presented by Patrick Celis on 29 May 2018

10 Project Brazilian Regulatory Framework for ATMP rules to accelerate the approval process of rare disease products, priority products for the Public Health System and also for special situations in the absence of therapeutic alternatives. The discussion on advanced therapy products in Brazil is providing a reflection on the traditional regulatory instruments for medicines and health products. For example: - on conditional marketing authorization, - simplification of analysis processes, - approved clinical trials based risk assessment.

11 Challenges Develop Technical Guides (Standards) Accessible publications of regulatory decisions Prospective international harmonization and convergence of regulatory approaches Regulatory environment for ATMPs is dynamic, complex and advancing fast! Continual need to have current regulatory intelligence Early, frequent and appropriate interactions between regulatory agencies and researchers/producers is highly recommended!!!!

12 How to make? ANVISA s key principles: based on a regulatory network, collective decision making, transparency, supporting innovation. A clear regulatory framework for ATMP: Gene therapy, cell therapy and tissue Strong support for ATMPs: CAT Early access tools, for example, ATMP classification, risk based approach, conditional marketing authorization Engaging with ANVISA: presubmission meetings. Early engagement encouraged for ATMPs.

13 Agência Nacional de Vigilância Sanitária - Anvisa SIA Trecho 5 - Área especial 57 - Lote 200 CEP: Brasília - DF Telefone: