12 April 2017 EMA/699038/2016 Inspections, Human Medicines Pharmacovigilance & Committees
CAT work plan 2017 – adopted by the Committee on 12 April 2017
Table of Content 1. Evaluation activities for human medicines .............................................. 2 1.1. Pre-authorisation activities .................................................................................... 2 1.2. Initial-evaluation activities ..................................................................................... 4 1.3. Other specialised areas and activities ...................................................................... 6
2. Horizontal activities and other areas ..................................................... 10 2.1. Committees and working parties .......................................................................... 10
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1. Evaluation activities for human medicines 1.1. Pre-authorisation activities 1.1.1. Revision of ATMP specific guidelines Key objectives •
Revision of the Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells.
Activities in 2017 – 2019 CAT activities to achieve the objectives set for this area: •
Finalise a concept paper on the revision of the above mentioned guideline, to be published by end of 3Q 2017
•
Publication of the draft of the revised Guideline for external consultation by 1Q 2018
•
Finalise the Revision of the Guideline by 4Q 2018
CAT will collaborate with the BWP for the revision of the quality part of this guideline. CAT topic leader: Marcos Timón Other Committee participants: Member/alternate
Name
MS
CAT topic leader
Marcos Timon
Spain
Member
Ilona Reischl
Austria
Member
Paula Salmikangas
Finland
Member
Christiane Niederlaender Belaid Sekkali
UK
Portugal
Expert
Margarida Menezes Ferreira Tiina Palomäki
Expert
Guido Pantè
Italy
Expert
Matthias Renner
Germany (PEI)
Expert
Brigitte Anliker
Germany (PEI)
Expert
Nicoloas Anagnou
Greece
Alternate Alternate
CAT work plan 2017 EMA/699038/2016
Belgium
Finland
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1.1.2. Development of ATMP specific guidelines Key objectives •
Development of a guideline on comparability of cell-based medicinal products
Activities in 2017-2019 CAT activities to achieve the objectives set for this area: •
Finalise a concept paper on the above mentioned guideline, to be published by end 4Q 2017
•
Publication of the draft of the Guideline for external consultation by 4Q 2018
•
Finalise the Guideline in 2019
CAT will collaborate with the BWP for the development of the quality part of this guideline. CAT topic leader: Margarida Menezes-Ferreira / Ilona Reischl Other Committee participants: Member/alternate
Name
MS
CAT topic leader
Margarida Menezes-Ferreira
Portugal
CAT topic leader
Ilona Reischl
Austria
Member
Violaine Closson Carella
France
Member
Paula Salmikangas
Finland
Member
Maura O’Donovan
Ireland
Member
Tomáš Boráň
Czech Republic
Member
Marc Turner
Member
Bernd Gänsbacher
Alternate
Belaid Sekkali
Scottish National Blood Transfusion Service Institute of experimental oncology and therapeutics Belgium
Alternate
Ivana Haunerova
Czech Republic
Expert
Tiina Palomäki
Finland
Expert
Louise Bisset
UK
Expert
Thomas Hinz
Germany (PEI)
CAT work plan 2017 EMA/699038/2016
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1.2. Initial-evaluation activities 1.2.1. Reflection on the Benefit Risk assessment of ATMPs During the multi-stakeholder meeting of 27 May 2016 on Advanced therapy medicinal products, stakeholders noted that current practice of defining the benefit-risk balance focuses mainly on risks and called for additional emphasis to be placed on expected but realistic benefits, particularly where patients have incurable diseases or where suitable treatments are lacking. CAT will reflect how this observation can be taken into account during the benefit-risk assessment that forms the basis of the approval of a marketing authorisation for ATMPs.
Key objectives •
Reflection on the Benefit-Risk assessment of ATMPs
Activities in 2017 CAT activities to achieve the objectives set for this area: •
Hold a meeting with the CAT interested parties to initiate the reflection on benefit-risk criteria specific to ATMPs. Deadline: 4Q 2017
•
Based on the outcome of the interested parties’ meeting, discuss the possibility and need to develop a CAT discussion paper on benefit-risk assessment of ATMPs. Deadline: 4Q 2017
•
If the development of a discussion paper on the benefit-risk assessment of ATMPs is decided, include this topic as a formal CAT activity in CAT work plan for 2018. CAT will collaborate with the CHMP, PRAC, PCWP and HCPWP for the development of this discussion paper.
CAT topic leader: Martina Schüßler-Lenz Other Committee participants: Member/alternate
Name
MS
CAT topic leader
Martina Schüßler-Lenz
Germany
Member
Claire Beuneu
Belgium
Member
Kieran Breen
Member
Maura O’Donovan
European Parkison’s Disease Association Ireland
Member
Bernd Gänsbacher
Member
Marc Turner
Alternate
Erik Briers
Alternate
Michelino Lipucci di Paola
CAT work plan 2017 EMA/699038/2016
Institute of experimental oncology and therapeutics Scottish National Blood Transfusion Service Europa Uomo – The European Prostate Cancer Coalition European Organisation for Rare Diseases (Eurordis)
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1.2.2. Reflection on the use of Registry data for the initial evaluation of ATMPs and during the post-authorisation phase. Key objectives •
CAT reflection on the use of registry data for the approval and post-marketing follow-up of ATMPs
Activities in 2017 CAT activities to achieve the objectives set for this area: •
Hold a meeting with the CAT interested parties to initiate the reflection on the use of registry data for the approval and post-marketing follow-up of ATMPs. Deadline: 4Q 2017
•
Based on the outcome of the interested parties’ meeting, discuss the possibility and need to develop a CAT discussion paper on the use of registry data for the approval and post-marketing follow-up of ATMPs. Deadline: 4Q 2017
•
If the development of a discussion paper on the above is decided, include this topic as a formal CAT activity in CAT work plan for 2018. CAT will collaborate with the CHMP for the development of this discussion paper.
CAT topic leader: Kieran Breen Other Committee participants: Member/alternate
Name
MS
CAT topic leader
Kieran Breen
Member
Tomáš Boráň
European Parkinson’s Disease Association Czech Republic
Member
Simona Badoi
Romania
Member
Marc Turner
Member
Mariette Driessens
Alternate
Erik Briers
Alternate
Michele Lipucci
Scottish National Blood Transfusion Service European Genetic Alliances Network (EGAN) Europa Uomo – The European Prostate Cancer Coalition Eurordis
Alternate
Angeliki Roboti
Greece
Expert
Klaus Rensing
Germany (PEI)
CAT work plan 2017 EMA/699038/2016
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1.3. Other specialised areas and activities 1.3.1. Scientific and Regulatory considerations on gene editing technologies Key objectives •
Reflection on the regulatory status of medicines based on, or produced by means of gene editing technologies
•
Consideration of the scientific requirements of medicines based on, or produced by means of gene editing technologies
Activities in 2017-2018 CAT activities to achieve the objectives set for this area: •
Discussion on the regulatory status of medicines based on, or produced by means of gene editing technologies. Deadline: 3Q 2017
•
Develop a CAT document on the regulatory considerations of this new technology (for use within the EU network). Deadline 4Q 2017
•
Organise an expert meeting to discuss with developers / experts in the field scientific challenges of this new technology, with the involvement of members from the Biologics Working Party (BWP) and the Pharmacogenomic Working Party (PGWP). Deadline 4Q 2017
•
Prepare a meeting report, publication or CAT document on regulatory and scientific considerations related to the development of medicines based on or produced by means of gene editing technologies. Deadline: 3Q 2018.
CAT topic leader: Paolo Gasparini Other Committee participants: Member/alternate
Name
MS
CAT topic leader
Paolo Gasparini
Italy
Member
Toivo Maimet
Estonia
Member
Paula Salmikangas
Finland
Member
Asterios Tsiftsoglou
Greece
Member
Maura O’Donovan
Ireland
Member
Dariusz Sladowski
Poland
Member
Simona Badoi
Romania
Member
Metoda Lipnik-Stangelj
Slovenia
Member
UK
Member
Christiane Niederlaender Bernd Gänsbacher
Alternate
Belaid Sekkali
Alternate
Portugal
Alternate
Margarida MenezesFerreira Egbert Flory
Alternate
Rune Kjeken
Norway
Alternate
Marcos Timon
Spain
Alternate
Michele Lipucci
Eurodis
CAT work plan 2017 EMA/699038/2016
Institute of experimental oncology and therapeutics Belgium
Germany
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Member/alternate
Name
MS
Expert
Tiina Palomäki
Finland
Expert
Silke Schuele
Germany (PEI)
CAT work plan 2017 EMA/699038/2016
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1.3.2. Considerations on novel scientific and regulatory approaches for making ATMPs more readily available to patients Key objectives •
Reflection and horizon scanning on novel approaches to make ATMPs more readily available to patients, such as the use of adeno-associated viral vectors
Activities in 2017 CAT activities to achieve the objectives set for this area: •
A CAT subgroup will initiate discussion and prepare a draft document with examples of such approaches and challenges related to them, for discussion in the CAT plenary meeting or during a CAT Strategic Review and Learning meeting. Deadline: 4Q 2017
•
Identify issues that require further reflection and plan ahead for activities to be explored in the CAT 2018 Work Plan. CAT will collaborate with the BWP and the HMA innovation network on this topic. Deadline: 4Q 2017.
•
Organisation of an expert meeting on scientific consideration for adeno-associated vector (AAV) based GTMPs. Deadline: 4Q 2017.
CAT topic leader: Hans Ovelgönne / Rune Kjeken Other Committee participants: Member/alternate
Name
MS
CAT topic leader
Hans Ovelgönne
Netherlands
CAT topic leader
Rune Kjeken
Norway
Member
Paula Salmikangas
Finland
Member
Toivo Maimets
Estonia
Member
Paolo Gasparini
Italy
Member
Simona Badoi
Romania
Member
Metoda Lipnik-Stangelj
Slovenia
Member
Christiane Niederlaender
UK
Member
Bernd Gänsbacher
Member
Marc Turner
Alternate
Margarida Menezes-Ferreira
Institute of experimental oncology and therapeutics Scottish National Blood Transfusion Service Portugal
Contribute to the European Commission in support of the discussions on GMO related issues in the ad-hoc group of national experts on medicines and experts from the environmental authorities.
Activities in 2017-2018 CAT activities to achieve the objectives set for this area: •
Provide technical and scientific input to the Commission. Deadline: 2Q 2018
CAT topic leader: Ilona Reischl Other Committee participants: Member/alternate
Name
MS
CAT topic leader
Ilona Reischl
Austria
Member
Paula Salmikangas
Finland
Member
Claire Beuneu
Belgium
Member
Violaine Closson Carella
France
Member
Krisztian Fodor
Hungary
Member
Dariusz Sladowski
Poland
Alternate
Margarida Menezes Ferreira
Portugal
Alternate
Marcos Timon
Spain
Alternate
Erik Briers
Europa Uomo - The European Prostate Cancer Coalition
CAT work plan 2017 EMA/699038/2016
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2. Horizontal activities and other areas 2.1. Committees and working parties 2.1.1. Setting up of an Ad hoc COMP-CAT working group Key objectives •
Explore the possibility to set up and define the activities of an Ad hoc COMP-CAT working group
Activities in 2017 CAT activities to achieve the objectives set for this area: •
Initiate discussion with COMP colleagues on areas for collaboration between COMP and CAT on orphan ATMPs. Deadline 3Q 2017
•
Discuss in CAT and COMP the possibility to set up an Ad hoc working group as a format to facilitate the interaction between the two Committees. Deadline 4Q 2017
CAT topic leader: Simona Badoi Other Committee participants: Member/alternate
Name
MS
CAT topic leader
Simona Badoi
Romania
Member
Mariëtte Driessens
Member
Kieran Breen
Expert
Guido Pantè
European Genetic Alliances Network (EGAN) European Parkinson's Disease Association Italy
CAT work plan 2017 - European Medicines Agency - Europa EU
Apr 12, 2017 - Member. Marc Turner. Scottish National Blood Transfusion. Service .... CAT will collaborate with the BWP and the HMA innovation network on this ... 1.3.3. Addressing the Environmental Risk assessment of ATMPs containing.
Jan 26, 2017 - different aspects, or if these aspects call for the development of new ... Joint EMA Symposium in the margins of the CEN-ISBS conference, ...
Feb 15, 2017 - Improve the quality of initial orphan designation applications by ... Maintenance ... Development of strategies to implement recommendations ...
Jan 27, 2017 - 30 Churchill Place â Canary Wharf â London E14 5EU â United Kingdom. An agency of the European Union. Telephone +44 (0)20 3660 .... best available scientific expertise in the Network and ensure that paediatric information related
Dec 15, 2016 - Work plan for the Radiopharmaceutical Drafting Group for. 2017. Chairperson: Anabel Cortes Blanco. Status of the work plan: Adopted in ...
5 days ago - Guidelines should include more details on the principles of good information design in which content and layout are ... relevance and importance of the QRD template is also acknowledged in this respect as it is the main tool .... databas
Jan 31, 2018 - Activities in 2018. PRAC activities to achieve the objectives set for this area: â¢. Review of lessons learnt from the pilot regulatory network study involving EMA, Spain and the United Kingdom, as a source of learnings for regulatory
Dec 14, 2017 - Send a question via our website www.ema.europa.eu/contact ... ad-hoc to respond to time-sensitive requests on products and to progress guidelines, as required. 2. Guidelines. 2.1. New EU Guidelines ... Recommendation to the CAT on data
Jul 5, 2017 - Send a question via our website www.ema.europa.eu/contact ... non-proprietary names (INN) and therapeutic areas for all new ... also available in the monthly reports of the Committee for Orphan Medicinal Products (COMP).
May 8, 2017 - Information Management Division ... This document provides current information related to the volume and evaluation of .... Plasma master file.
Jan 30, 2018 - HMPC work plan. EMA/HMPC/150152/2018. Page 2/7. Evaluation activities for herbal medicinal products as defined in Reg. (EC) No 726/2004 and Dir. 2001/83/EC. 1.1. Establishment and update of EU herbal monographs and list entries. Activi
Feb 13, 2017 - Monthly report on application procedures, guidelines and .... of MRLs for new substances under article 3 of Regulation (EC) No 470/2009.
Oct 9, 2017 - In 2017, an audit of the EU Portal and Database will take place and, on the basis of .... These frameworks collectively offer a platform for exchange and multi-stakeholder dialogue at the. European ...... EudraVigilance database and by
Oct 13, 2017 - applications for initial evaluations, extensions, variations and renewals concerning marketing authorisations (MAs); ... Initial evaluation of marketing authorisation applications. 2014. 2015. 2016 .... Out of scope. 1 Establishment of
Mar 16, 2017 - Information Management Division ... This document provides current information related to the volume and evaluation of .... Plasma master file.
Oct 9, 2017 - possible expertise for the regulation of medicines in the European Union (EU). To deliver on its responsibilities, EMA works closely with the NCAs. This means the environment, trends, workload forecasts and implementation of a number of
Jul 7, 2017 - COMMUNITY MARKETING AUTHORISATIONS AND EXTENSIONS. 2.1. Opinions ... Quality. Rapp: E. Werner .... solutions table with EMA and CVMP responses to the recommendations made by the FishMed Plus. Coalition ...
Jun 22, 2016 - Information Management Division ... This document provides current information related to the volume and evaluation of .... Plasma master file.
Feb 9, 2017 - Notification of discontinuation of a paediatric development which is covered by an agreed ... for the following reason(s): (tick all that apply).
Jul 4, 2016 - Send a question via our website www.ema.europa.eu/contact ... non-proprietary names (INN) and therapeutic areas for all new ... also available in the monthly reports of the Committee for Orphan Medicinal Products (COMP).