EU Regulatory Network To form a critical view on the current and - - PDF document

SLIDE 1

EMEA/CHMP EMEA/CHMP-

• Think Tank Group on Innovation, Research

Think Tank Group on Innovation, Research and Drug Development and Drug Development

Xavier Luria, MD Head of Safety and Efficacy of Medicines, H- Pre Unit EMEA

EU Regulatory Network Challenges and Opportunities for Croatia Rijeka, November 2008

EMEA-CHMP Think-Tank group on Innovation

To form a critical view on the current and future scientific development

• f medicinal products and related regulatory standards in Europe:

To identify bottlenecks in the development of medicinal products To identify possible redundant or irrelevant regulatory requirements To discuss the application of new methods and procedures in the development and regulation of medicinal products To support European Commission in Innovative Medicines Initiative To facilitate discussions between CHMP, academia and industry on innovative approaches for the development of medicinal products

Participants of the EMEA-CHMP Think-Tank group

• Daniel Brasseur, Chair
• Gonzalo Calvo Rojas
• Bruno Flamion, Vice Chair
• Bengt Danielsson
• Robert Hemmings
• Pekka Kurki
• Ingemar Persson
• Patrick Salmon
• Cristina Sampaio
• Bo Aronsson
• Emer Cooke
• Xavier Kurz
• Patrick Le Courtois
• Xavier Luria
• Outi Maki-Ikola, Project

Manager

• Marisa Papaluca-Amati
• John Purves
• Agnes Saint-Raymond
• Spiros Vamvakas
• Meetings with 14 pharmaceutical companies

11/2005-2006

• Meetings with academic groups 4Q/2006:

– EASD (European Association for the Study of Diabetes) EFNS (European Federation of Neurological Societies) – EORTC (European Organisation for Research and Treatment of Cancer) – ESCMID (European Society for Clinical Microbiplogy and infectious Diseases)

• Final Report: March 2007

http://www.emea.europa.eu/pdfs/human/itf/12731807en.pdf

EMEA-CHMP Think-Tank group on Innovation

• 1. Communication and interaction with regulators

during the lifecycle of the products

• 2. Global harmonisation
• 3. Emerging science for clinical development and

regulatory approval 3.a Translational development, clinical methodology 3.b Advanced Therapies

• 4. Pharmacovigilance
• 5. Guidelines
• 6. Antimicrobials

EMEA-CHMP Think-Tank group on Innovation

• Areas for improvement in the EMEA/CHMP operations

– Communication during the lifecycle of the product – Guidelines

• Scientific topics

– Biomarkers – Statistical methods and clinical study designs – Faster access tools – Risk Management Plans

• Areas for the attention of the EC and NCAs in the EU

Network

– Guidelines for Clinical trials – Global aspects – Interaction between Industry and Academia – Advanced Therapies

• Special public health topic: Antimicrobial resistance

EMEA-CHMP Think-Tank group on Innovation

SLIDE 2

1. Interactions with stakeholders: in depth and well prepared discussion meetings 2005-2006

• «signals» for action

undertaken immediately by the EMEA 2. Think-tank Report published March 2007 3. Plan for implementation agreed by the CHMP Dec 07 4. EMEA Executive launches an EMEA-wide process improvement project (ongoing) 5. CHMP work programme 2008-2010

Which approach has been taken for implementation Which approach has been taken for implementation Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Which approach has been followed for implementation Which approach has been followed for implementation

Actions undertaken within the frame of the EMEA improvement Actions undertaken within the frame of the EMEA improvement exercise for operations in the area of exercise for operations in the area of Human medicines Human medicines – – Improved procedures Improved procedures

• OMP: better use of resources

OMP: better use of resources

• SA: implementation pre

SA: implementation pre-

• submission, shorter timelines

submission, shorter timelines and extended involvement of expertise via IT methods and extended involvement of expertise via IT methods

• Pharmacovigilance referrals: streamline of procedures

Pharmacovigilance referrals: streamline of procedures and better information to the public and better information to the public

• Plasma master files: better information

Plasma master files: better information

• Pharmacovigilance: improvement in signal detection

Pharmacovigilance: improvement in signal detection – – Improved CHMP and working parties meetings management Improved CHMP and working parties meetings management

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

1.

• 1. Areas for action

Areas for action with with general general impact impact IMI, interaction IMI, interaction with with networks, modernisation of networks, modernisation of working working and and communication communication methods methods 2.

• 2. Communication and interaction during the lifecycle of the produ

Communication and interaction during the lifecycle of the products cts Early dialogue on innovation, scientific advice, gap between SA Early dialogue on innovation, scientific advice, gap between SA and MAA and MAA 3.

• 3. Global harmonisation

Global harmonisation ICH, Bilateral arrangements ICH, Bilateral arrangements

Areas for action agreed with the CHMP (1) Areas for action agreed with the CHMP (1) Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

4. 4. Emerging science for clinical development and regulatory approva Emerging science for clinical development and regulatory approval l

• Support to early clinical development, translational medicine

Support to early clinical development, translational medicine

• Innovative R&D methods including biomarkers, new imaging

Innovative R&D methods including biomarkers, new imaging techniques, modelling and simulation, new statistical techniques, modelling and simulation, new statistical approaches approaches

• F

Faster access tools: conditional approval and accelerated aster access tools: conditional approval and accelerated review review

• Advanced therapies

Advanced therapies 5. 5. Pharmacovigilance Pharmacovigilance 6. 6. Guidelines Guidelines 7. 7. Antimicrobials Antimicrobials

Areas for action agreed by the CHMP (2) Areas for action agreed by the CHMP (2) Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

EMEA Policy on the participation in Innovative Medicines Initiat EMEA Policy on the participation in Innovative Medicines Initiative ive (IMI) (IMI) – – research projects published May 2008) research projects published May 2008) http://www.emea.europa.eu/pdfs/24929308en.doc http://www.emea.europa.eu/pdfs/24929308en.doc

In response to this paper 10 consortia requested EMEA to participate

Advisory body for decisions established (SMO) Advisory body for decisions established (SMO)

EMEA partner of a consortium EMEA partner of a consortium EMEA leading an applicant consortium to address the call for EMEA leading an applicant consortium to address the call for “ “Strengthening the monitoring of Benefit Strengthening the monitoring of Benefit-

• Risk

Risk” ” Making partnership to happen and establishing the grounds for Making partnership to happen and establishing the grounds for transparent and fair future involvement: requests for participat transparent and fair future involvement: requests for participation to ion to consortia have now their pathway. consortia have now their pathway.

• 1. Innovative Medicines Initiative
• 1. Innovative Medicines Initiative

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

• 1. Interaction with networks
• 1. Interaction with networks

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

ENCePP network : phase 1 of the project completed. Five ENCePP

working groups active. Representatives of learned societies members

• f the ENCePP Advisory Board

Paediatric network: implementation strategy published Jan 08; to foster high quality ethical research on medicinal products to be used in children, strengthening scientific, technical and/or administrative competences in the performance of paediatric clinical trials

• Coordination group to start in 2009

New process for Learned societies and stakeholders active consultation on EMEA scientific work: database and procedures being established (MIS)

• 2009

Activities of the HCP and PO working group: reinforced contribution to EMEA activities in 2009

SLIDE 3

• 1. Modernisation of
• 1. Modernisation of working

working and communication and communication methods methods

• WEB Sharing and video/

WEB Sharing and video/Tc Tc conferencing implemented in CHMP, conferencing implemented in CHMP, SAWP and increasingly in other working parties to SAWP and increasingly in other working parties to optimise

• ptimise

availability of expertise availability of expertise

• Training sessions on key areas reinforced by the use of IT medi

Training sessions on key areas reinforced by the use of IT media a (e.g. (e.g. Paediatrics Paediatrics and and ATPs ATPs) )

• Paperless working method (MMD)

Paperless working method (MMD) succesfully succesfully implemented in implemented in CHMP and extending to working parties (e.g. SAWP, BWP) CHMP and extending to working parties (e.g. SAWP, BWP)

• eCTD

eCTD submission of applications implementation plan (ongoing) submission of applications implementation plan (ongoing)

• EMEA scientific publication policy and publication review group

EMEA scientific publication policy and publication review group established May 08 established May 08

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

EMEA opportunities for dialogues: Regulatory and procedural guidance (EMEA web page) http://www.emea.europa.eu/htms/human/raguidelines/intro.htm Task Force (ITF), Briefing Meetings Paediatrics Small and Medium-sized Enterprises (SME) Orphans Scientific Advice and Protocol Assistance Pre-Marketing Authorisation, Pre-Submission Application & Evaluation, Post-Opinion Post-Marketing Authorisation

2.

• 2. Communication in the lifecycle of the products

Communication in the lifecycle of the products Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Scientific advice: further improvements Scientific advice: further improvements

Peer review Peer review by CHMP: by CHMP: introduced mid 2007 (positive impact in 30% of the cases). To be consolidated in 2009 End End-

• of
• f-
• phase 2 advices

phase 2 advices: “guaranteed” discussion meeting with SAWP in case of divergent views EMEA/FDA SA EMEA/FDA SA: available but no requests received from sponsors New procedures for New procedures for minor SA follow minor SA follow-

• up/clarifications

up/clarifications: : to be formalised in 2009

2.

• 2. Communication in the lifecycle of the products

Communication in the lifecycle of the products Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action From Scientific advice to Marketing Authorisation From Scientific advice to Marketing Authorisation Application: Filling the gap between SA and MAA Application: Filling the gap between SA and MAA

SAWP: Discussion on gaps detected in the available data when preparing phase III advice EMEA: Pre-submission meetings for MAA with extended discussions with the scientific secretariat and Rapporteurs up to 2 years before filing MAA: implementation in 2009

2.

• 2. Communication in the lifecycle of the products

Communication in the lifecycle of the products Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Bilateral arrangements with FDA Bilateral arrangements with FDA

Collaboration with FDA ongoing on established clusters (e.g. paediatrics, OMPs, oncology, pandemic flu, pharmacogenomics, qualification of biomarkers) New initiatives being undertaken in the area of ATPs Initial steps taken in the area of nanomedicines (for further action in 2009). Bilateral EC-EMEA/FDA will define the new clusters

3.

• 3. Global Harmonisation

Global Harmonisation Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action New bilateral confidentiality arrangements New bilateral confidentiality arrangements

Bilateral arrangements with Japan and Canada in the implementation planning phase Exchange of officials and visits with FDA and PMDA MHLW Identification of a new function in Directorate and Executive to support for international liaison

3.

• 3. Global Harmonisation

Global Harmonisation Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

SLIDE 4

Maintain the focus on ICH activities Maintain the focus on ICH activities

ICH Q8 Annex Pharmaceutical Development – step 3 comments by May 08 ICH Q9 Quality Risk Management: planning EU implementation ICH Q10 Pharmaceutical Quality System: planning EU implementation ICH S2 genotoxicity guideline - rev.1comments by May 08 ICH S6 - Preclinical Safety Evaluation of Biotechnology-Derived Products ICH M3 (Non-Clinical Safety Studies for the Conduct of Human Clinical Trials for Pharmaceuticals – rev. 2 comments by Oct 08 ICH E2F Development safety update reports (comments by Dec 08) E7 studies in elderly: concept paper for revision adopted by SC ICH E16: PG biomarker qualification dossier structure and data format Gene Therapy: oncolytic viruses and shedding studies reflection papers

3.

• 3. Global Harmonisation

Global Harmonisation Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action EMEA promote dialogue on innovative R&D EMEA promote dialogue on innovative R&D

Workshops on scientific priority areas identified: Workshops on scientific priority areas identified:

2006 2006: Design Space, Slowing the Progression of Neurodegenerative : Design Space, Slowing the Progression of Neurodegenerative Diseases, Diseases, Biomarkers Biomarkers 2007 2007: Workshops on requirements for : Workshops on requirements for first first-

• in

in-

• man clinical trials

man clinical trials for for potential high potential high-

• risk medicinal products, Joint with EC workshop on clinical

risk medicinal products, Joint with EC workshop on clinical trials, trials, Immunogenicity Assessment of Therapeutic Proteins, Cell based Immunogenicity Assessment of Therapeutic Proteins, Cell based medicines, Gene medicines, Gene therapy vectors shedding, therapy vectors shedding, adaptive design in confirmatory trials adaptive design in confirmatory trials 2008 2008: Neurodegenerative Diseases : Neurodegenerative Diseases-

• "Focus on Dementia"

"Focus on Dementia" , , Modeling Modeling in paediatric in paediatric medicines, Brain Diseases in the Elderly, Clinical Outcome Measu medicines, Brain Diseases in the Elderly, Clinical Outcome Measures and res and endpoints for efficacy assessment in spinal muscular atrophy, endpoints for efficacy assessment in spinal muscular atrophy, Integrating PG Integrating PG BM BM Early into Drug Development: Early into Drug Development: PK as a working example PK as a working example 2009 2009 : : New New statistical statistical methods methods

• 4. Science in clinical development and regulatory approval
• 4. Science in clinical development and regulatory approval

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Translational medicine Translational medicine

Biomarkers Biomarkers Global collaboration: Global collaboration:

• ngoing with FDA, planned in 2009 with PMDA and Health Canada
• ngoing with FDA, planned in 2009 with PMDA and Health Canada

Participation in consortia (IMI, C Participation in consortia (IMI, C-

• Path):

Path): EMEA/CHMP actively EMEA/CHMP actively involved in C involved in C-

• Path PSTC consortium

Path PSTC consortium Workshops: Workshops: 1 1st

st held in 2005 and 2

held in 2005 and 2nd

nd in 2006

in 2006 Pilot BM qualification process in 2007 Pilot BM qualification process in 2007 Formal BM qualification process implemented in 2008 Formal BM qualification process implemented in 2008

• 4. Science in clinical development and regulatory approval
• 4. Science in clinical development and regulatory approval

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Translational medicine Translational medicine

Non Non-

• clinical requirements

clinical requirements to support early phase I clinical trials:

to support early phase I clinical trials: One workshop and two guidelines (for high One workshop and two guidelines (for high-

• risk medicinal product and for

risk medicinal product and for gene therapy) completed. ICH M3 revision gene therapy) completed. ICH M3 revision

Modelling and simulation Modelling and simulation

Workshop on Workshop on Pk Pk modelling for paediatric medicinal products held in 2008 modelling for paediatric medicinal products held in 2008 Discussion forum, global, with regulators, academia and industry Discussion forum, global, with regulators, academia and industry, to be , to be established: CHMP work established: CHMP work-

• program project, to be developed in 2009

program project, to be developed in 2009-

• 2010

2010 Additional expertise for scientific evaluation and creation of n Additional expertise for scientific evaluation and creation of networks of etworks of excellence should be considered. excellence should be considered.

• 4. Science in clinical development and regulatory approval
• 4. Science in clinical development and regulatory approval

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Clinical methodology Clinical methodology Statistical aspects /new study designs Statistical aspects /new study designs

Flexible design and Flexible design and bayesian bayesian approach discussed in the EWP approach discussed in the EWP Reflection Paper on Methodological Issues in Confirmatory Clinic Reflection Paper on Methodological Issues in Confirmatory Clinical al Trials planned with an adaptive design Trials planned with an adaptive design http://www.emea.europa.eu/pdfs/human/ewp/245902enadopted.pdf CHMP CHMP workprogramme workprogramme project: establishing in 2008 a group with project: establishing in 2008 a group with statistical expertise: statistical expertise: Support a consistent science-based approach Workshops Training Specialised network

• 4. Science in clinical development and regulatory approval
• 4. Science in clinical development and regulatory approval

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Faster access tools Faster access tools

Conditional approval and RMP: implemented in scientific advice a Conditional approval and RMP: implemented in scientific advice and nd final opinions final opinions Accelerated review: CHMP work programme project 2008 Accelerated review: CHMP work programme project 2008-

• 2010:

2010: Analysis of the experience Analysis of the experience Identify obstacles Identify obstacles Propose measures to improve success of the provisions Propose measures to improve success of the provisions

• 4. Science in clinical development and regulatory approval
• 4. Science in clinical development and regulatory approval

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

SLIDE 5

Advanced Therapies

New Guidelines/ Reflection papers/ Question and Answer New Guidelines/ Reflection papers/ Question and Answer-

• documents

documents Contribution to the EC implementation plans Contribution to the EC implementation plans Classification and certification of quality and non Classification and certification of quality and non-

• clinical data

clinical data for for SMEs SMEs Rolling scientific evaluation Rolling scientific evaluation CHMP and CAT CHMP and CAT

• 4. Science in clinical development and regulatory approval
• 4. Science in clinical development and regulatory approval

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

• 4. Science in clinical development and regulatory approval
• 4. Science in clinical development and regulatory approval

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Benefit/Risk assessment Benefit/Risk assessment

Reflection Paper On Benefit Reflection Paper On Benefit-

• Risk Assessment Methods In The

Risk Assessment Methods In The Context Of The Evaluation Of Marketing Authorisation Application Context Of The Evaluation Of Marketing Authorisation Applications s Of Medicinal Products For Human Use Of Medicinal Products For Human Use

• To revise the current benefit

To revise the current benefit-

• risk assessment section of the

risk assessment section of the CHMP assessment report templates, incorporating a structured lis CHMP assessment report templates, incorporating a structured list t

• f benefit and risk criteria and guidance
• f benefit and risk criteria and guidance
• To further research into methodology of benefit risk assessment

To further research into methodology of benefit risk assessment, , involving further experts and assessors involving further experts and assessors

Guideline on safety and efficacy follow Guideline on safety and efficacy follow-

• up/risk management of

up/risk management of ATMPs ATMPs; ; http://www.emea.europa.eu/pdfs/human/advancedtherapies/14999508e http://www.emea.europa.eu/pdfs/human/advancedtherapies/14999508en.pdf n.pdf Facilitate access to Facilitate access to Eudravigilance Eudravigilance data/ signal detection tools; data/ signal detection tools;

• Guideline on the Use of Statistical Signal Detection Methods in

Guideline on the Use of Statistical Signal Detection Methods in the the EudraVigilance EudraVigilance Data Analysis System (EMEA/106464/2006/Rev.1): adopted by CHMP Data Analysis System (EMEA/106464/2006/Rev.1): adopted by CHMP June 2008 June 2008 http://www.emea.europa.eu/pdfs/human/phvwp/10646406enfin.pdf http://www.emea.europa.eu/pdfs/human/phvwp/10646406enfin.pdf

• Access on communication signals: reflection stage

Access on communication signals: reflection stage European network of centres for European network of centres for pharmacoepidemiology pharmacoepidemiology and and pharmacovigilance pharmacovigilance:

: Advisory Committee and 5 working groups established

Advisory Committee and 5 working groups established. . Next meeting Oct.08 Next meeting Oct.08

Update risk management system Update risk management system-

• guideline: Action 2009

guideline: Action 2009 Workshop on effectiveness of Workshop on effectiveness of RMPs RMPs: Action 2009 : Action 2009

• 5. Pharmacovigilance
• 5. Pharmacovigilance

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

New, quicker Question and Answer New, quicker Question and Answer-

• documents: i

documents: implemented in a

mplemented in a number of areas to supplement guidelines or to focus on specific number of areas to supplement guidelines or to focus on specific issues. issues. Examples include: Examples include: Question & Answers on the CHMP Guideline on the Limits of Question & Answers on the CHMP Guideline on the Limits of Genotoxic Genotoxic Impurities Impurities http://www.emea.europa.eu/pdfs/human/swp/43199407en.pdf http://www.emea.europa.eu/pdfs/human/swp/43199407en.pdf Questions & Answers on the use of Cocktail studies for investiga Questions & Answers on the use of Cocktail studies for investigating in vivo ting in vivo drug interaction potential drug interaction potential http://www.emea.europa.eu/pdfs/human/ewp/49078407en.pdf http://www.emea.europa.eu/pdfs/human/ewp/49078407en.pdf

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

• 6. Guidelines
• 6. Guidelines
• 6. Guidelines
• 6. Guidelines

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action Consultation of experts and stakeholders Consultation of experts and stakeholders

Implemented via public workshops on key principles at very early Implemented via public workshops on key principles at very early stage stage and/or and/or prior finalisation to address major comments prior finalisation to address major comments

• Priorities for guideline development on the basis of the needs o

Priorities for guideline development on the basis of the needs of f product development to be established in consultation with product development to be established in consultation with stakeholders stakeholders

• Reconsider the balance between general guidance (retrospective)

Reconsider the balance between general guidance (retrospective) and and individual (prospective) advices individual (prospective) advices

Issues under consideration in the dedicated project of the CHMP Issues under consideration in the dedicated project of the CHMP working programme 2008 working programme 2008-

• 2010

2010

Gap analysis on current medical needs for Gap analysis on current medical needs for antimicrobials, priority list of pathogens antimicrobials, priority list of pathogens

Joint CHMP/PDCO WG with ECDC + Collaboration established with Joint CHMP/PDCO WG with ECDC + Collaboration established with Duke University and REACT and with ESCMID (European Society) Duke University and REACT and with ESCMID (European Society)

• agreement on pathogens priorities for drug development

agreement on pathogens priorities for drug development

• establishment of search criteria and searches run on databases

establishment of search criteria and searches run on databases to identify to identify products in development (Phase I to III plus pre products in development (Phase I to III plus pre-

• registration)

registration)

• rating agreed and being assigned to products that cover any of

rating agreed and being assigned to products that cover any of the priority the priority pathogens pathogens

Tailor Tailor-

• made requirements to encourage development of new

made requirements to encourage development of new antimicrobials (4 antimicrobials (4th

th Q 2009)

Q 2009) Initiatives for incentives for developing old antimicrobials or Initiatives for incentives for developing old antimicrobials or niche niche product (4 product (4th

th Q 2009)

Q 2009)

• 7. Antimicrobials
• 7. Antimicrobials

Think Think-

• tank

tank’ ’s: recommendations for action s: recommendations for action

SLIDE 6

• Implementation

Implementation of action in all areas

• f action in all areas identified

identified by the by the Think Think Tank has Tank has major impact on EMEA major impact on EMEA operations

• perations and

and resources resources

• Work

Work in in progress progress or

• r clearly

clearly planned planned for for most most TT TT recommendations recommendations

• Ongoing

Ongoing efforts to efforts to integrate integrate synergies synergies among among all all stakeholders stakeholders involved involved

• Implementation

Implementation to to take take in to in to account account experience experience, , evolution evolution of the

• f the

needs needs and the and the changing changing pharmaceutical pharmaceutical environment environment (e.g. new (e.g. new legal legal provisions) provisions)

CONCLUSIONS

Thanks for your attention!

! xavier.luria@emea.europa.eu xavier.luria@emea.europa.eu

BACK UP SLIDES

• 1. Communication and interaction with regulators

Briefing meetings to be further developed Faster scientific advice SA peer review Pre-submission meetings for MAA with extended scientific discussions

• 2. Global harmonisation

Routine discussions with other regulators (FDA) on biomarkers, new statistical approaches, risk management plans, advanced therapies, parallel SA

• 3. Emerging science

3.a Translational development, clinical methodology Involvement in the US Critical path Initiative and support to the European IMI, workshops and training, reflection papers, early involvement in biomarker development and acceptance, further resources on statistical methods, links with European and international statisticians, EWP subgroup on adaptive designs and other statistical aspects. 3.b Advanced Therapies New Guidelines/ Reflection papers/ Question and Answer documents, contribution to the EC implementation plans, informal dialogue opportunities, rolling scientific evaluation and certification of quality and non-clinical data for SMEs

• 4. Pharmacovigilance

Update risk management system-guideline Encourage SA on risk management plans Analyse and develop principles for acceptable standards in risk minimization activities Workshop on effectiveness of RMPs Facilitate access to Eudravigilance data/ signal detection tools, Establish European network of centres for pharmacoepidemiology and pharmacovigilance

• 5. Guidelines

New, quicker Question and Answer-documents More Reflection Papers More focussed guidance Specific assessor networks Prioritisation + early consultation of experts and stakeholders guideline on drug diagnostic co- development

SLIDE 7

• 6. Antimicrobials

Gap analysis on current medical needs for antimicrobials priority list of pathogens: joint CHMP/PDCO WG with ECDC to define and supervise its progress Tailor-made requirements to encourage development of new antimicrobials