Regulatory Experience for a Gene Therapy Product in a Large Phase II European Study. Disclaimer
|
|
- Maximilian Clark
- 5 years ago
- Views:
Transcription
1 Regulatory Experience for a Gene Therapy Product in a Large Phase II European Study Catherine Mathis Director of Regulatory Affairs transgene 1 Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to Drug Information Association, Inc. ( DIA ), its directors, officers, employees, volunteers, members, chapters, councils, Special Interest Area Communities or affiliates, or any organization with which the presenter is employed or affiliated. These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United Statest of America and other countries. ti Used by permission. All rights reserved. Drug Information Association, DIA and DIA logo are registered trademarks or trademarks of Drug Information Association Inc. All other trademarks are the property of their respective owners
2 Agenda Regulatory framework for gene therapy products Definitions Legal Texts CTA process for GTMPs Experience of phase II studies Conclusions 3 3 Agenda Regulatory framework for gene therapy products Definitions Legal Texts CTA process for GTMPs Experience of phase II studies Conclusions 4 4 2
3 Regulatory Framework: Definitions 2009/120/EC: Gene Therapy Medicinal Product (GTMP) Biological medicinal product with the following characteristics: recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence; therapeutic, prophylactic or diagnostic effect related directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence. GTMPs shall not include vaccines against infectious diseases. 5 5 Regulatory Framework: Definitions GTMPs include: naked nucleic acid complex nucleic acid or non viral vectors viral vectors genetically modified cells In Europe, GTMPs are Medicinal Products and Genetically Modified Organisms (GMOs) 6 6 3
4 Regulatory Framework: Definitions Separate therapeutic applications using GTMPs: Inserting a corrective gene, repairing a mutated gene, regulating a gene Genetic diseases Integrative viral vectors used for the gene transfer: retrovirus, lentivirus 7 7 Regulatory Framework: Definitions Expression of immunotherapeutic genes: «therapeutic vaccines» Oncology and infectious disease fields Tumor antigen (like MUC1, CEA), human virus antigens (HCV, HPV, HIV), cytokines (IL2, IFNg, GM-CSF)» MOA: induce cellular immune responses (CTL) against the tumor or the infectious agents Suicide gene (HSV-TK + ganciclovir)» MOA: transformation of prodrug into cytotoxic drug 8 8 4
5 Regulatory Framework: Definitions Today, no GTMPs on the market in EU and/or USA Two GTMPs approved in China Veterinary field: Several vaccines based on the same technology on the market for many years: e.g. Raboral, Eurifel (Merial) 9 9 Agenda Regulatory framework for gene therapy products Definitions Legal Texts CTA process for GTMPs Experience of phase II studies Conclusions
6 Regulatory Framework: Legal texts All GTMPs are regulated by: the EU pharmaceutical regulation: 2001/83/EC Directive Regulation (EC) No 726/2004 Advanced Therapies Regulation (N 1394/2007) with in addition the environmental component regulated by EU GMO Directives 1990/219/EC (2009/41/EC) and 2001/18/EC Directives Regulatory Framework: Legal texts Directive 2009/41/EC repealing 90/219/EC and 98/81/EC related to the confined use of GMO Classification of operations: Type A for R D and non-commercial purposes Type B for any operation other than a Type A operation GMO classification: Group I for non-pathogenic and Group II for the others Class of risk related to the pathogenicity: Class 1, 2, 3 or 4: risk nul (L1), low (L2), moderate (L3) or high (L4) Description of Confinement Biosafety Level: L1, L2, L3, L4: takes into account the facilities organisation, the equipment and the good practices of work
7 Regulatory Framework: Legal texts Directive 1990/220/EC abrogated by the Directive 2001/18/EC Related to the dissemination of GMO and risk for environment: deliberate release Define the risk assessment, a better access of the information to the public and a better traceability of the products Agenda Regulatory framework for gene therapy products Definitions Legal Texts CTA process for GTMPs Experience of phase II studies Conclusions
8 Regulatory Framework: CTA process For any clinical trial with GTMPs: CTA submitted to the Competent t Authorities (CA): Health Authorities + National Biosafety Authorities follow the same format as for any IMPs: IMPD (2001/20/EC) Technical dossier on the GMO: Annex II and III conform to 2001/18/EC: environmental risk assessment (ERA) Specific questionnaire or other documents specific to each EU country Regulatory Framework: the CTA process Clinical site: Must be authorised for GMO manipulation Specific documentation to be submitted to the local and/or national Biosafety Authorities by the clinical site Descriptive documentation of the GMO, facilities description and responsible people Ethics: Protocol to be authorised by national and possibly local Ethic Committees Protocol +ICF + IB + Insurance to be submitted
9 Regulatory Framework: the CTA process GMO specific documentation: Annex III related to the ERA Information relating to the GMO Information relating to the conditions of release and the receiving environment Information relating to the interactions between the GMOs and the environment Information on monitoring, control and treatment of waste and emergency response plans Regulatory Framework: the CTA process e.g. in France Sponsor: EudraCT number + Dossier > 6 months process Haut Conseil des Biotechnologies (Research and Environment Authorities) - GMO classification - Risk assessment - Confined use or deliberate release AFSSAPS Unité Essai Clinique Evaluation by the Gene Therapy Group of Afssaps Central Ethics Committee (Comité de Protection des Personnes) GMO facilities approval Authorization Opinion Clinical sites Start of the Clinical trial
10 Regulatory Framework: the CTA process e.g. in Germany Clinical sites Sponsor: EudraCT number + Dossier > 6 months process GMO facilities approval Landesamt für Gesundheit und Soziales / Bezirksregierung (Research and Environment Authorities) - GMO and facilities i classification - Risk assessment - Confined use or deliberate release PEI Evaluation by the Dept of Clinical Trials (Dept. Klinische Prüfungen) Authorization Ethic Committee (central + local) Opinion Start of the Clinical trial Agenda Regulatory framework for gene therapy products Definitions Legal Texts CTA process for GTMPs Experience of phase II studies Conclusions
11 Experience of phase II studies Transgene focused on: development of immunotherapeutic ti products based on recombinant viral vectors technology Two recombinant viral vector platforms: Vaccinia virus with the use of the highly attenuated MVA strain (Modified Vaccinia virus Ankara) Adenovirus type Experience of phase II studies The product: TG4010 Recombinant vaccinia virus of the MVA strain expressing MUC1 tumor antigen and human IL2 SC route, dose of 10e8 pfu, one injection every week for 6 wks then every 3 wks up to progression The targeted diseases: Cancer diseases expressing MUC1: NSCLC, prostate, breast cancers, RCC
12 Experience of phase II studies Mechanism of action: Stimulation of the patient s immune system with induction of a Th1 immune response expected to eliminate tumor cells expressing MUC1 TG4010 aims at increasing survival, decreases tumor burden and progression with minimal toxicity, in the treatment of NSCLC and potentially other MUC1 expressing tumors TG4010 TH Dendritic cell CD4 CD8 NK Antibody TH2 B adc IFNγ IL-2 Activated Dendritic Cell Adapted from Nature Reviews Immunology Experience of phase II studies Phase II program with TG Phase II studies from 2002 to 2008 > 300 patients in around 60 sites in 7 countries: France, Germany, Poland, Hungary, Belgium, USA, Switzerland UK+Czech Republic: withdrawn
13 Experience of phase II studies UK withdrawn Belgium Germany Czech R withdrawn Hungary Poland France M0 M1 M2 M3 M4 M5 M6 M7 M8 M9 M10 M11 M12 M13 M14 M15 Months First patient in CTA submission and approval by the HA EC submission and approval byec Experience of phase II studies Country TTA Regulatory bodies (CA, EC, Clinical site Biosafety) France 4 Centralized process to the CA Site approved for GMO: for pharmaceutical and. simple and quick process <2mo biosafety Logistical aspects for GMO manipulation: slow down the process Some fear from medical staff due to neg impact of agriculture GMO Poland 11 Contracts signed prior to reg Site approved for GMO: submission. long process, technical dossier, Quick process with CA and EC new field for PI, Long process for biosafety: no existing committee TTA: Time To Approval in months
14 Experience of phase II studies Country TTA Regulatory bodies (CA, EC, Biosafety) Germany 7 Quick process with CA Site approval for GMO needed prior CA approval Longer process with EC: local and leading Belgium 4 Clear regulatory process Easy contact with the Authorities Site approval for GMO needed prior CA approval Clinical site Site approved for GMO by the «Lander» authority:. long process, technical dossier, new field for PI if no previous exp. Site approved for GMO:. long process, technical dossier, new field for PI if no previous exp. TTA: Time To Approval in months Experience of phase II studies Country TTA Regulatory bodies (CA, EC, Clinical site Biosafety) Hungary 4 Simple and rapid regulatory process: centralized to the NIP EC advice given to the NIP No direct contacts between EC and sponsor No specific approvals for GMO 5 mo from regulatory approval and first patient in: for contractual issues with the central Lab TTA: Time To Approval in months
15 Experience of phase II studies Country TTA Regulatory bodies (CA, EC, Biosafety) Czech Republic WD Positive and quick approval from CA Biosafety:. additional viral shedding data requested: not justified, CTA withdrawn UK WD CMC questions from CA Conditional approval from GTAC with some questions Not evaluated by the local REC Clinical site Independent Biosafety expert to certify the conformity of the clinical sites: report to be provided to the Biosafety Authorities Request for approval from local biosafety committee (GMSC). No GMSC on site: long process to put it in place Decision to withdraw the CTA TTA: Time To Approval in months WD: Withdrawn Agenda Regulatory framework for gene therapy products Definitions Legal Texts CTA process for GTMPs Experience of phase II studies Conclusions
16 Conclusions The past (> 10 years ago): Study generally limited to small size/monocenter Requirements not in line with large study e.g. patient confinement requested (up to 17 days confinement until absence of any viral particles in the biological fluids) No clear regulatory framework for GMO: lack of guidances, not the same level of information to be submitted, no timelines, lack of expertise Today: Conclusions More experience and expertise from the industry and the CA Improvement in the regulatory submission process with better harmonization of the documentation to be submitted (EU 2001/20) More guidances in place to better define the regulatory framework of GTMPs
17 BUT Conclusions Still lack of harmonisation for CTA particularly in the case of GTMPs different national dossier requirements discrepant application of Community law. Significant administrative burden specifically for GTMPs for the sponsor and the investigators: compliance with national and local biosafety requirements Conclusions Solutions Administrative i ti simplication still needed d Limited re-discussion of issues at different levels: national CA, EC, local EC Simplification of the approval process of the clinical protocol amendments To include the GMO related assessments (risk and clinical sites) in the CTA for centralized review by the Medicinal Agencies
18 Conclusions Europe must become more attractive and more competitive in the development of innovative IMPs Thank you for your attention
Advanced-therapy medicinal products: new competencies in hospital pharmacy Seminar PH4. Relevant Financial Relationships - None
EAHP March 2016 Advanced-therapy medicinal products: new competencies in hospital pharmacy Seminar PH4 Dr. Lenka Taylor Pharmacy University Hospital Heidelberg Disclosure Relevant Financial Relationships
More informationDevelopment Stage of Therapeutic Vaccines: The Regulator s View
Development Stage of Therapeutic Vaccines: The Regulator s View Thomas Hinz Head, Section Therapeutic Vaccines Paul Ehrlich Institute, Germany thomas.hinz@pei.de 1 DISCLAIMER This is the personal views
More informationImpact of the transposition of the European Clinical Trials Directive. CEMO, Paris 17 November 2004
Impact of the transposition of the European Clinical Trials Directive CEMO, Paris 17 November 2004 Dr Martine Dehlinger-Kremer VP Regulatory Affairs International Agenda Overview of key areas of Directive
More informationEU Perspective on Regulatory Issues for Biologics
Oncology Biologics Development Primer 29 February 2008 Robert Charnas Disclosure Employee and shareholder of Amgen, Inc. Thousand Oaks, CA Worked in Basel, Switzerland for 19 Years and dealt with European
More informationENVIRONMENTAL RISK ASSESSMENTS: EU CLINICAL TRIALS WITH GMO-BASED ATMPS ANN GORMAN, AMGEN LTD, UK 16 DECEMBER 2016
ENVIRONMENTAL RISK ASSESSMENTS: EU CLINICAL TRIALS WITH GMO-BASED ATMPS ANN GORMAN, AMGEN LTD, UK 16 DECEMBER 2016 SIMPLIFIED DEFINITIONS Genetically Modified Organism (GMO)? An organism, e.g virus, plant,
More informationICH Considerations. Oncolytic Viruses September 17, 2009
INTERNATIONAL CONFERENCE ON HARMONISATION OF TECHNICAL REQUIREMENTS FOR REGISTRATION OF PHARMACEUTICALS FOR HUMAN USE ICH Considerations Oncolytic Viruses September 17, 2009 1. Introduction Oncolytic viruses
More informationICH Considerations on Viral/Vector Shedding; and Overview of Gene Therapy Activity in Canada
ICH Considerations on Viral/Vector Shedding; and Overview of Gene Therapy Activity in Canada Anthony Ridgway, Ph.D. Senior Regulatory Scientist Biologics & Genetic Therapies Directorate Health Canada Open
More informationClinical Trials Environment EU Legislation: Ausblick auf die neue Gesetzgebung für klinische Prüfungen
The Future of the Regulatory Berlin, 31.05.2013 Clinical Trials Environment EU Legislation: Qualifizierungstag für Study Nurses Ausblick auf die neue Gesetzgebung für klinische Prüfungen Dr. med. Ingrid
More informationICH CONSIDERATIONS Oncolytic Viruses
European Medicines Agency Pre-authorisation Evaluation of Medicines for Human Use 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 ICH CONSIDERATIONS Oncolytic Viruses 20 November 2008 EMEA/CHMP/GTWP/607698/2008
More informationICH Considerations Oncolytic Viruses ONCOLYTIC VIRUSES (EMEA/CHMP/ICH/607698/2008) TRANSMISSION TO CHMP November 2008
European Medicines Agency October 2009 EMEA/CHMP/ICH/607698/2008 ICH Considerations Oncolytic Viruses ONCOLYTIC VIRUSES (EMEA/CHMP/ICH/607698/2008) TRANSMISSION TO CHMP November 2008 TRANSMISSION TO INTERESTED
More informationProcedure and experience in Germany
www.pei.de National Competent Authorities experience with Environmental Risk Assessment (ERA) before setting clinic trials Procedure and experience in Germany Brigitte Anliker 5th Annual Regulatory Conference
More informationGMO Technology Conference
GMO Technology Conference The regulation of Clinical Trials on humans involving therapies containing or consisting of genetically modified organisms The Printworks, Dublin Castle 10 th & 11 th October
More informationConsiderations on regulatory aspects
Considerations on regulatory aspects Regulatory framework for medicinal products in the context of therapeutic use of bacteriophages EMA Workshop on 8 June Presented by Zigmars Sebris on 8 June 2015 Regulatory
More informationOVERVIEW OF DIRECTIVE 2001/20. Paul Derbyshire. Background & History. Aims of Directive 2001/20
OVERVIEW OF DIRECTIVE 2001/20 Paul Derbyshire Background & History CONDUCT OF TRIALS III/3976/88 (July 1991) ICH/135/95 (January 1997) 2001/20 75/318 Q,S,E Testing Part 4B: GCP 91/507 MEDICINAL PRODUCTS
More informationREQUEST FOR AUTHORISATION TO THE COMPETENT AUTHORITY: REQUEST FOR OPINION OF THE ETHICS COMMITTEE:
Annex 1: Clinical trial Application Form REQUEST FOR AUTHORISATION OF A CLINICAL TRIAL ON A MEDICINAL PRODUCT FOR HUMAN USE TO THE COMPETENT AUTHORITIES AND FOR OPINION OF THE ETHICS COMMITTEES IN THE
More informationRegulatory requirements for cell based medicinal products
Regulatory requirements for cell based medicinal products 資料 3-2 Committee 25 August 2010 Dr. Bettina Klug, MSc Paul-Ehrlich-Institut, Langen klube@pei.de Paul-Ehrlich-Institut Federal Institute for Vaccines
More informationFolder Name Documents included Explanation
Structure for the documents to be submitted in the case of electronic submission of applications for clinical trials at the BfArM (Federal Institute for Drugs and Medical Devices) / Paul-Ehrlich Institut
More informationClinical Trials application process, legislation & guidelines
Clinical Trials application process, legislation & guidelines IMB Clinical Trials Seminar 19 th June 2012 Elaine Breslin MB BCh (NUI), PhD, FRCPI Clinical Assessment Manager 19/06/2012 Slide 1 IMB Mission
More information-Regulation (EC) No.1394/2007 -Regulation (EC) No. 668/2009
Introduction to Advanced Therapy Medicinal Products Regulation -Regulation (EC) No.1394/2007 -Regulation (EC) No. 668/2009 -Directive 2009/120/EC Dr. Maura O Donovan F.R.C.O.G. MA MD M.R.C.P.I. CAT member
More informationNew Regulation in Japan and Future Direction of PMDA. Tatsuya Kondo, M.D., Ph.D. Chief Executive Pharmaceuticals and Medical Devices Agency (PMDA)
New Regulation in Japan and Future Direction of PMDA Tatsuya Kondo, M.D., Ph.D. Chief Executive Pharmaceuticals and Medical Devices Agency (PMDA) 1 Disclaimer The views and opinions expressed in the following
More informationContent. Introduction: Approval of ATMPs. Support to ATMP developers. Role of EMA and National Competent Authorities
Content Introduction: Role of EMA and National Competent Authorities Approval of ATMPs What are ATMPs? Centralised procedure Support to ATMP developers 1 Introductory statements This talk is on marketing
More informationGuidance for applicants for ethics committee opinion on the conduct of a clinical trial of pharmaceuticals
KLH-EC-01 APPLICATION FOR ETHICS COMMITTEE OPINION ON THE CONDUCT OF A CLINICAL TRIAL IN THE CZECH REPUBLIC requirements governing the documentation to be submitted This guideline is being published in
More informationPer Bergman Head of the GMO Unit. GMO conference Brussels, September 2009
EU Risk Assessment of GMOs Roles of EFSA, Member States and European Commission Per Bergman Head of the GMO Unit GMO conference Brussels, 14-15 September 2009 GMO regulation in EU In the EU, products that
More informationRSC/CT Det. no. 1/2013
RSC/CT Det. no. 1/2013 Protocol 1631-P The English version of this Determination was prepared in order to help comprehension by non-italian mother tongue users, but is NOT an official document. Please
More informationA SHORT GUIDE TO THE PROCEDURE FOR A CLINICAL TRIAL APPLICATION IN THE KINGDOM OF BAHRAIN
A SHORT GUIDE TO THE PROCEDURE FOR A CLINICAL TRIAL APPLICATION IN THE KINGDOM OF BAHRAIN Version 1 - June 2017 A Short Guide For CT Application 1 2 A Short Guide For CT Application DEFINITIONS Clinical
More informationCOMMISSION DIRECTIVE 2009/120/EC
15.9.2009 Official Journal of the European Union L 242/3 DIRECTIVES COMMISSION DIRECTIVE 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC of the European Parliament and of the Council on
More informationThe Regulatory Environment for Therapeutic Vaccines. Thomas Hinz Head, Section Therapeutic Vaccines Paul Ehrlich Institute, Germany
The Regulatory Environment for Therapeutic Vaccines Thomas Hinz Head, Section Therapeutic Vaccines Paul Ehrlich Institute, Germany hinth@pei.de 1 Topics Addressed Regulatory environment - EU - Germany
More informationEMA s role & responsibility for the development of modern/advanced therapies
EMA s role & responsibility for the development of modern/advanced therapies Agenda Current regulatory picture Overall Regulatory framework New Committee at EMA Current Activities & Challenges Egbert Flory
More informationGuide to EU Clinical Trial Application Form
Deleted: Guide to EU Clinical Trial Application Form SCOPE This user guide has been prepared to help applicants complete the application form to the HPRA for authorisation of a clinical trial
More informationCurrent Issues in EU Cell & Gene Therapy Regulation. Christiane Niederlaender, UK CAT Delegate, MHRA
Current Issues in EU Cell & Gene Therapy Regulation Christiane Niederlaender, UK CAT Delegate, MHRA.CASSS C> June 2018 Disclaimer The views expressed in this presentation are my personal views and may
More information27 September Introduction
27 September 2017 Possible solutions to improve the European regulatory procedures for clinical trials with Advanced Therapy Medicinal Products consisting of or containing Genetically Modified Organisms
More informationExplanatory note on general fees payable to the European Medicines Agency
Explanatory note on general fees payable to the European Medicines Agency The fees, fee exemptions and definitions described in this explanatory note apply as of 1 June 2017 and are based on Council Regulation
More informationImpact of Clinical EU Directive on the implementation of Early development studies in Europe
Impact of Clinical EU Directive on the implementation of Early development studies in Europe Alain Patat, M.D. Translational Development Wyeth Research Paris, France AGAH-Club Phase 1 1 st Syposium Strasbourg
More informationProject Management Standards Applied to Complex Clinical Trials. Disclaimer
Project Management Standards Applied to Complex Clinical Trials Alexander Gissler, PMP ProjectPharm Ltd., Owner & Director 1 Disclaimer The views and opinions expressed in the following PowerPoint slides
More informationComments on: Name of Organisation
Comments on: THE RULES GOVERNING MEDICINAL PRODUCTS IN THE EUROPEAN UNION VOLUME 10 - GUIDANCE DOCUMENTS APPLYING TO CLINICAL TRIALS HARMONISED REQUIREMENTS FOR NON INVESTIGATIONAL MEDICINAL PRODUCTS IN
More informationREGISTRATION DOCUMENT FOR RECOMBINANT DNA RESEARCH
EHRS Date Received: Reg. Doc. No.: REGISTRATION DOCUMENT FOR RECOMBINANT DNA RESEARCH Principal Investigator: Penn ID#: Position Title: School: Department: Mailing Address: Mail Code: Telephone: FAX: E-mail:
More informationExplanatory note on general fees payable to the European Medicines Agency
Explanatory note on general fees payable to the European Medicines Agency The fees, fee exemptions and definitions described in this explanatory note apply as of 1 June 2017 and are based on Council Regulation
More informationPharmacovigilance in Asia: The China Perspectives. Disclaimer
Pharmacovigilance in Asia: The China Perspectives Rebecca Wang, MD, FRCP, FACC Head, Product Development Drug Safety Operation, Asia Pacific Roche Shanghai Disclaimer The views and opinions expressed in
More informationEARLY DRUG DEVELOPMENT CONSULTANCY & SERVICES CLINICAL RESEARCH SOLUTIONS
EARLY DRUG DEVELOPMENT CONSULTANCY & SERVICES CLINICAL RESEARCH SOLUTIONS As a full service contract research organisation performing phase I to IV clinical trials across Europe and the Americas for 40
More informationPhase Appropriate GMPs for IMPs. Presented by: Karen S. Ginsbury For: IFF, October 31, Nov 02, 2017
Phase Appropriate GMPs for IMPs Presented by: Karen S. Ginsbury For: IFF, October 31, Nov 02, 2017 1 Lets start with References https://mhrainspectorate.blog.gov.uk/2016/0 5/20/manufacture-of-investigationalmedicinal-products-frequently-askedquestions/
More informationCOMMITTEE FOR VETERINARY MEDICINAL PRODUCTS (CVMP)
The European Agency for the Evaluation of Medicinal Products EMEA/CVMP/550/02-FINAL COMMITTEE FOR VETERINARY MEDICINAL PRODUCTS (CVMP) GUIDELINE ON REQUIREMENTS FOR CONCURRENT ADMINISTRATION OF IMMUNOLOGICAL
More informationPMDA Perspectives on Companion Diagnostics Development in Japan. Reiko Yanagihara, Ph.D. Deputy Review Director Office of In Vitro Diagnostics, PMDA
PMDA Perspectives on Companion Diagnostics Development in Japan Reiko Yanagihara, Ph.D. Deputy Review Director Office of In Vitro Diagnostics, PMDA 1 Disclaimer Content Slide The views and opinions expressed
More informationIncorporating Risk- Based Monitoring Strategies: Challenges in Implementation Sherri Hubby, Director, U.S. Quality Assurance
Incorporating Risk- Based Monitoring Strategies: Challenges in Implementation Sherri Hubby, Director, U.S. Quality Assurance 26th Annual EuroMeeting 25-27 March 2014 ACV, Vienna Austria. Disclaimer The
More informationHorizon 2020 Health Summit-IIS La Fe. Valencia, 12 de diciembre de 2017
Horizon 2020 Health Summit-IIS La Fe Valencia, 12 de diciembre de 2017 2 History 3 AsphaTeam +75 members Consultants with Life Science Background 20 team members with PhD High knowhow in drug development
More informationNew Regulation for Advanced Therapies including Oncology Biological Products. isbtc Global Regulatory Summit
New Regulation for Advanced Therapies including Oncology Biological Products isbtc Global Regulatory Summit Patrick Celis, PhD European Medicines Agency (EMEA) Presentation Overview EMEA and the European
More informationCOMMENTS FROM EUROPABIO GENERAL COMMENTS
SUBMISSION OF COMMENTS ON DETAILED GUIDANCE FOR THE REQUEST FOR AUTHORISATION OF A CLINICAL TRIAL ON A MEDICINAL PRODUCT FOR HUMAN USE TO THE COMPETENT AUTHORITIES, NOTIFICATION OF SUBSTANTIAL AMENDMENTS
More informationFelicia Favorito Clinical Operations Leader TESARO Inc.
Felicia Favorito Clinical Operations Leader TESARO Inc. Navigating your way around protocols which intervene with country specific regulations to ensure valuable time isn t lost during the clinical trial
More informationAPPLICATION FORM. Application for a Recognised Research Ethics Committee (REC) Opinion on a Clinical Trial on a Medicinal Product for Human Use.
APPLICATION FORM Application for a Recognised Research Ethics Committee (REC) Opinion on a Clinical Trial on a Medicinal Product for Human Use. Form 1 This application form should be completed and submitted
More informationClinical trial applications in the EU and US
Clinical trial applications in the EU and US Alain Patat, M.D. Translational Development Wyeth Research Paris, France AGAH-Club Phase 1 1 st Symposium Strasbourg 17-18 March 2005 Overview of the presentation
More informationExplanatory note on fees payable to the European Medicines Agency
30 March 2015 EMA/167155/2014 Executive Director Explanatory note on s payable to the European Medicines Agency The s, exemptions and definitions described in this Explanatory Note apply as of 1 April
More informationUpdate from the Center for Biologics Evaluation and Research (CBER) Peter Marks, M.D., Ph.D. GMP By The Sea 2017
Update from the Center for Biologics Evaluation and Research (CBER) Peter Marks, M.D., Ph.D. GMP By The Sea 2017 Outline Products regulated Significance of complex biologics Product and process Cutting
More informationLabelling Harmonization 2011
Labelling Harmonization 2011 DIA, Bethesda, MD Core Labeling During Clinical Development Su Yueh Lin Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual
More informationEUROPEAN COMMISSION ENTERPRISE DIRECTORATE-GENERAL
EUROPEAN COMMISSION ENTERPRISE DIRECTORATE-GENERAL Single market : management & legislation for consumer goods Pharmaceuticals : regulatory framework and market authorisations Brussels, ENTR/CT 5.2 Final
More informationIndividualised medicine: regulatory challenges
www.pei.de Individualised medicine: regulatory challenges Outline Revision IVD Directive Opportunities and challenges Possibilities for interaction http://upload.wikimedia.org/wikipedia/commons/thumb/6/66/gummy_bears.jpg
More informationThe new EU clinical trial regulation 536/2014 : Low interventional trials
The new EU clinical trial regulation 536/2014 : Low interventional trials KCE Greet Musch Brussels, 28 th November 2017 Agenda: 1: Legal framework EU regulation 536/2014 Q&A document : algorithm 2: Risk
More informationThe EU Regulatory Framework for the ATMP
The EU Regulatory Framework for the ATMP Disclaimer I attend this course as an individual expert, and do not represent EMA or ISS. The views expressed here are my personal views, and may not be understood
More informationAPPLICATION FOR PAEDIATRIC INVESTIGATION PLAN / WAIVER
Version-number 3.0.0 APPLICATION FOR PAEDIATRIC INVESTIGATION PLAN / WAIVER This application is submitted for a: Medicinal Product for which the Applicant does not hold a marketing authorisation in the
More informationNAS and KAS Industry perspective
NAS and KAS Industry perspective Henk Schuring Group Vice President Regulatory Affairs Europe Genzyme Europe BV, Netherlands 26th Annual EuroMeeting 25-27 March 2014 ACV, Vienna Austria Disclaimer The
More informationCHANGES TO A GENE THERAPY MEDICINAL PRODUCT: REGULATORY VIEWS
CHANGES TO A GENE THERAPY MEDICINAL PRODUCT: REGULATORY VIEWS Maria Cristina Galli, Ph.D. Istituto Superiore di Sanità Roma, Italy EATRIS ATMP platform chair ISCT 2016 Global Regulatory Perspective Workshop
More informationRegulatory perspective to review MRCT data for drug approval: Focusing on ethnic factor evaluation
Regulatory perspective to review MRCT data for drug approval: Focusing on ethnic factor evaluation Dr Yoshiaki Uyama Pharmaceuticals and Medical Devices Agency (PMDA) 1 Disclaimer The views and opinions
More informationANNEX. CHAPTER I General principles
ANNEX REGULATIONS on the authorisation by the NMA of clinical trials/notification to the National Medicines Agency of non-interventional studies on medicinal products for human use in Romania CHAPTER I
More informationHTAs and EMA working together: 23 parallel scientific advice procedures later - what have we learned?
HTAs and EMA working together: 23 parallel scientific advice procedures later - what have we learned? DIA 26th Annual EuroMeeting, Vienna 2014 Jan Regnstrom, MD, PhD Senior Scientific Officer An agency
More informationImpact of MRCT after ICH E17 fully implement -Industry perspective-
Impact of MRCT after ICH E17 fully implement -Industry perspective- 5th Joint Conference of Taiwan and Japan on Medical Products Regulation December 1st, 2017 Chikara Kikuchi Vice-Chairperson, Drug Evaluation
More informationCOMMITTEE FOR VETERINARY MEDICINAL PRODUCTS NOTE FOR GUIDANCE 1 : DNA VACCINES NON-AMPLIFIABLE IN EUKARYOTIC CELLS FOR VETERINARY USE
The European Agency for the Evaluation of Medicinal Products Evaluation of Medicines for Veterinary Use CVMP/IWP/07/98-FINAL COMMITTEE FOR VETERINARY MEDICINAL PRODUCTS NOTE FOR GUIDANCE 1 : DNA VACCINES
More informationREGISTRATION DOCUMENT FOR RECOMBINANT & SYNTHETIC DNA RESEARCH
IBC Date Received: Reg. Doc. No.: REGISTRATION DOCUMENT FOR RECOMBINANT & SYNTHETIC DNA RESEARCH Principal Investigator: --------------------------------------- Position Title: -------------- Department:
More informationMultinational Clinical Trials in EU How NCAs can improve the system? Dr C. Bélorgey, Head of Clinical Trials Department Afssaps Chair of CTFG
Multinational Clinical Trials in EU How NCAs can improve the system? Dr C. Bélorgey, Head of Clinical Trials Department Afssaps Chair of CTFG Bonn 30 April 2010 European CTs bodies (Competent Authorities)
More informationOral vaccines to protect patients against Clostridium difficile infection
Oral vaccines to protect patients against Clostridium difficile infection Jonathan Kearsey: Leads To Development Antibiotics and their alternatives-fixing and feeding the pipeline Project number: 601810
More informationOverview of ICH GTDG Activities and Current Topics in Gene Therapy
Overview of ICH GTDG Activities and Current Topics in Gene Therapy Klaus Cichutek Paul-Ehrlich-Institut, 63225 Langen, Germany www.biomed.brown.edu Chair, EMEA/CHMP GTWP Co-Chair ICH GTDG E-mail: cickl@pei.de
More informationBiosimilars China Guideline. Dr Dr Michel Mikhail
Biosimilars China Guideline Dr Dr Michel Mikhail 1 Contents Regulatory context of biologicals in China Decree 28 issued by SFDA October 2007 Proposed biosimilars guideline 10/29/14 Reference drugs to use
More informationExplanatory note on fees payable to the European Medicines Agency
09 December 2013 EMA/458574/2013 Executive Director Explanatory note on s payable to the European Medicines Agency The s, exemptions and definitions described in this Explanatory Note apply as of 1 January
More informationOrdinance on the Fees charged by the Swiss Agency for Therapeutic Products (Therapeutic Products Fees Ordinance)
Ordinance on the Fees charged by the Swiss Agency for Therapeutic Products (Therapeutic Products Fees Ordinance) of 2 December 2011 (Stand am 1. Januar 2015) The Agency Council of the Swiss Agency for
More informationProcedures in Regard to Entire Product Information Dr. Klaus Menges BfArM Bethesda (MD) October 14, 2011
Harmonization of Labeling Across Products with Same Active Moiety in the EU Procedures in Regard to Entire Product Information Texts Dr. Klaus Menges BfArM Bethesda (MD) October 14, 2011 Disclaimer The
More informationOUR MISSION OUR EXPERTISE OUR SERVICES
Capacités Biotherapeutics Solutions (C.B.S) is a newly created business unit of CAPACITÉS LLC, an affiliate company of the University of Nantes (France). By gathering experts and core facilities, C.B.S
More informationEUROPEAN COMMISSION HEALTH AND CONSUMERS DIRECTORATE-GENERAL
EUROPEAN COMMISSION HEALTH AND CONSUMERS DIRECTORATE-GENERAL Public health and Risk assessment Pharmaceuticals Brussels, 01 June 2010 SANCO/C/8/SF/dn D(2010) 326199 THE RULES GOVERNING MEDICINAL PRODUCTS
More informationBelgium, a European leader in clinical trials
Belgium, a European leader in clinical trials An attractive country for clinical trials to test innovative medicines In Europe it is nearly impossible to find other countries with as many clinical trials
More informationHow are medicines evaluated at the EMA
How are medicines evaluated at the EMA Presented by: Nathalie Bere Patient interaction / Stakeholders and communication Division An agency of the European Union The European System Centralised Procedure
More informationEU Clinical Trial Regulation A view from the Industry
Conference EU Clinical Trial Regulation EU Clinical Trial Regulation A view from the Industry Judith Creba, Executive Director, EU Regulatory Strategy, Novartis Pharma AG University of Basel, Law Faculty
More informationLabelling & Naming. European Biosimilars Group (EBG) perspective. >400 Million patient days worldwide clinical experience with EU biosimilar medicines
Labelling & Naming European Biosimilars Group (EBG) perspective >400 Million patient days worldwide clinical experience with EU biosimilar medicines Elke Grooten, Director Public Affairs Sandoz Europe
More informationRegulatory Affairs: Study Report of New Drug Registration Process in European Union
95 Review Article Regulatory Affairs: Study Report of New Drug Registration Process in European Union Yogeshkumar B. Viradiya*, Manoj B. Dagwar, Swapnil T. Lanjewar Department of Regulatory Affairs, Institute
More informationCMC Workshop CMC Challenges in Japan. April Bethesda, MD. Yoshihiro Matsuda, Ph.D., PMDA Session 10
CMC Workshop 2015 April 13-15 Bethesda, MD CMC Challenges in Japan Yoshihiro Matsuda, Ph.D., PMDA Session 10 Disclaimer The views and opinions expressed in the following PowerPoint slides are those of
More informationEUROPEAN COMMISSION ENTERPRISE AND INDUSTRY DIRECTORATE-GENERAL
EUROPEAN COMMISSION ENTERPRISE AND INDUSTRY DIRECTORATE-GENERAL Consumer goods Pharmaceuticals Brussels, [ ] ENTR/F/2/SF D(2009) [ ] Detailed guidance for the request for authorisation of a clinical trial
More informationUniversity of Delaware Department of Environmental Health & Safety Recombinant DNA Registration
Registration #: University of Delaware Department of Environmental Health & Safety Recombinant DNA Registration Directions: Please complete this form to register recombinant DNA research with the University
More information2016 Annual Meeting of Stockholders. October 20, 2016
2016 Annual Meeting of Stockholders October 20, 2016 Safe Harbor Statement Statements herein relating to future financial or business performance, conditions or strategies and other financial and business
More informationSyracuse University Institutional Biosafety Committee Protocol Application Form
Syracuse University Institutional Biosafety Committee Protocol Application Form The Syracuse University Institutional Biosafety Committee (IBC) has been established to protect the health of University
More informationMaster protocols: MHRA experience. Khadija Rantell
Master protocols: MHRA experience Khadija Rantell Disclaimer The views expressed in this presentation are those of the speaker and not necessarily those of the MHRA. 2 Life Sciences Industrial Strategy
More informationLondon, 11 October 2006 Doc. Ref. EMEA/CHMP/BWP/271475/2006 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP)
European Medicines Agency 1 2 London, 11 October 2006 Doc. Ref. 3 4 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) 5 DRAFT 6 7 GUIDELINE ON POTENCY TESTING OF CELL BASED IMMUNOTHERAPY MEDICINAL
More informationWhat is an ideal PSUR? A new focus based on aligned expectations
What is an ideal PSUR? A new focus based on aligned expectations Margarida Guimarães PRAC Member INFARMED, I.P. Periodic Safety Update Report Information Day 28 October 2016 London, UK Disclaimer The views
More informationAdvanced Therapies in Europe
Advanced Therapies in Europe 1 ATMPs in Europe (2009-2017) ~ 500 clinical trials using ATMPs in EU ~ 270 ATMP classifications 18 MAAs reviewed ~ 250 scientific advice requests 9 ATMPs approved 2 3 withdrawn
More informationCOMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP)
European Medicines Agency Pre-authorisation Evaluation of Medicines for Human Use London, 22 February 2006 EMEA/CHMP/BMWP/42832/2005 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) GUIDELINE ON SIMILAR
More informationGene therapy. Findings by Alert
Gene therapy Published Mar 21, 2000 Version 1 Findings by Alert Research in gene therapy has increased dramatically during the past 15 years, particularly in the United States. The research has encompassed
More informationHarmonized Quality Requirements for Biotech Investigational Medicinal Products - A Regulator's View
Harmonized Quality Requirements for Biotech Investigational Medicinal Products - A Regulator's View Brigitte Brake Pharmaceutical Biotechnology BfArM, Bonn Evaluation Criteria National vs. EU Clinical
More informationRegistration Document For Biohazards
Protocol #: Registration Document For Biohazards All applicants are required to complete the following sections: Principal Investigator Information Location of Study Section A: General Administrative Information
More informationProcess and guidelines for applications for approval of trials involving gene and other biotechnology therapies. November 2015
Process and guidelines for applications for approval of trials involving gene and other biotechnology therapies November 2015 HRC Gene Technology Advisory Committee For further information contact: Professor
More informationA. TRIAL IDENTIFICATION
PROTOCOL INFORMATION ON A CLINICAL TRIAL ON A MEDICINAL PRODUCT FOR HUMAN USE CONDUCTED IN A THIRD COUNTRY (i.e. a country outside of the EEA) Note: To ensure consistency the numbering of this form is
More informationQuality Assurance of Investigational Medicinal Products. Sue McKenzie PPQA (A)
Quality Assurance of Investigational Medicinal Products Sue McKenzie PPQA (A) Agenda for Today IMP Regulations - CTD and Annex 13 Role of QP for IMP Challenges of IMP Manufacture for the QP IMP Regulations
More informationTherapeutic Proteins BIT 230
Therapeutic Proteins BIT 230 CLOTTING Haemophilia Benefix Blood Products ANTICOAGULANT THROMBOLYTIC AGENTS tissue plasminogen activator streptokinase Coagulation pathway Factor VIII (Haemophilia A) Factor
More informationSharing Regulatory Intelligence: Best Practices and Case Studies
Sharing Regulatory Intelligence: Best Practices and Case Studies Carolyn Hynes Sr. Director, Global Regulatory Intelligence, GlaxoSmithKline 26th Annual EuroMeeting 25-27 March 2014 ACV, Vienna Austria
More informationReflection paper on classification of advanced therapy medicinal products
13 April 2012 Committee for Advanced Therapies (CAT). Reflection paper on classification of advanced therapy medicinal products Draft Agreed by CAT March 2012 Adoption by CAT for release for consultation
More informationConsiderations in Product Development with Advanced Therapies and Cancer Vaccines
Considerations in Product Development with Advanced Therapies and Cancer Vaccines Thomas Hinz Head of Section Therapeutic Vaccines Paul-Ehrlich-Institut hinth@pei.de Thomas Hinz, October 29, 2008, San
More informationCurrent Status of Gene Therapy Products in Japan
Current Status of Gene Therapy Products in Japan Division of Cellular and Gene Therapy Products National Institute of Health Sciences Eriko Uchida, Ph.D. Gene Therapy Direct application of gene therapy
More information