The Committee for Advanced Therapies (CAT) is responsible for assessing advanced therapy medicinal products (ATMPs) at the European Medicines Agency. The CAT evaluates ATMPs using centralized regulatory procedures. The CAT is composed of experts in various scientific areas relevant to advanced therapies like gene therapy, cell therapy, and tissue engineering. Patients' and healthcare professionals' organizations can also be represented on the CAT as full members to provide the patient perspective during the evaluation of ATMPs.
Genome editing by CRISPR systems has proven to be groundbreaking for basic biomedical research with significant implications for the treatment of human diseases. While the CRISPR-Cas9 and CRISPR-Cas12a (Cpf1) systems enable genome editing in a broad range of host species and cell types, both can exhibit poor editing efficiencies at specific target sites or in systems where delivery of CRISPR reagents is difficult. There are concerns about target specificity of the CRISPR-Cas9 system and, in many cases, typical remedies such as modified guide RNAs or mutant Cas9 proteins cause loss of genome editing efficiency. Many of these solutions for improving specificity were developed for delivery of the Cas9-gRNA complex via plasmid DNA vectors rather than delivery as ribonucleoproteins (RNPs). However, RNP delivery of CRISPR reagents is being increasingly used because of the risk of unwanted stimulation of the immune system by plasmid delivery.
In this webinar, Dr Vakulskas discusses improved Cas9 and Cas12a (Cpf1) nucleases that have been optimized to significantly increase editing efficiency in living cells. He also presents data showing that IDT’s latest high-fidelity Cas9, Alt-R HiFi S.p. Cas9 V3, increases on-target editing efficiency and dramatically reduces off-target editing.
i explained about basics of genome engineering and crispr system.
CRISPR will change the world and it is just the beginning, are you ready to meet the future? you think its great and beautiful or.....?
please give your feedback to my email
pooyanaghshbandi@yahoo.com
i am starting to write a critical and fantastic review article about CRISPR, if you are interested to join please contact me.
a brief description on the new emerging genome editing technology CRISPR-Cas9. this technique is making its place stronger and stronger day by day. and impossible things can be possible by this technique. and some main and famous names who discovered this technique.
Genome editing by CRISPR systems has proven to be groundbreaking for basic biomedical research with significant implications for the treatment of human diseases. While the CRISPR-Cas9 and CRISPR-Cas12a (Cpf1) systems enable genome editing in a broad range of host species and cell types, both can exhibit poor editing efficiencies at specific target sites or in systems where delivery of CRISPR reagents is difficult. There are concerns about target specificity of the CRISPR-Cas9 system and, in many cases, typical remedies such as modified guide RNAs or mutant Cas9 proteins cause loss of genome editing efficiency. Many of these solutions for improving specificity were developed for delivery of the Cas9-gRNA complex via plasmid DNA vectors rather than delivery as ribonucleoproteins (RNPs). However, RNP delivery of CRISPR reagents is being increasingly used because of the risk of unwanted stimulation of the immune system by plasmid delivery.
In this webinar, Dr Vakulskas discusses improved Cas9 and Cas12a (Cpf1) nucleases that have been optimized to significantly increase editing efficiency in living cells. He also presents data showing that IDT’s latest high-fidelity Cas9, Alt-R HiFi S.p. Cas9 V3, increases on-target editing efficiency and dramatically reduces off-target editing.
i explained about basics of genome engineering and crispr system.
CRISPR will change the world and it is just the beginning, are you ready to meet the future? you think its great and beautiful or.....?
please give your feedback to my email
pooyanaghshbandi@yahoo.com
i am starting to write a critical and fantastic review article about CRISPR, if you are interested to join please contact me.
a brief description on the new emerging genome editing technology CRISPR-Cas9. this technique is making its place stronger and stronger day by day. and impossible things can be possible by this technique. and some main and famous names who discovered this technique.
CRISPR is easily the best gene editing tool to date. For decades, scientists have been looking for a way to to perform precise changes to genetic sequences. In the past several years, researchers were able to exploit the immune systems of bacteria to edit the genome of other living cells. CRISPR is reported to have higher targeting efficiencies when compared to TALENs and Zinc Fingers. It is efficient, easy to use and cheap; making it a scalable genetic engineering tool that is highly desirable in various industry-wide applications.
Use of CRISPR-Cas9 has revolutionized targeted genome editing. However, rapid design of high-quality guide RNA (gRNA) sequences with high on-target and low off-target editing remains challenging. We implemented a machine learning algorithm to design high-quality gRNA sequences in 5 commonly used species (human, mouse, rat, zebrafish, and nematode). Our tool also designs gRNA sequences against custom targets, and can check existing gRNA designs for quality. In this webinar, we review our data illustrating this tool's performance and demonstrate its use in predicting and designing improved gRNAs for genome editing.
CRISPR/Cas9 gene editing is based on a microbial restriction system, that has been harnessed for genome targeting using only a short sequence of RNA as a guide.
The beauty of the system is that unlike protein binding based technologies such as Zinc Fingers and TALENs which require complex protein engineering, the design rules are very simple, and it is this fact that is allowing CRISPR to take genome engineering from a relatively niche persuit to the mainstream scientific community.
The principle of the system is that a short guide RNA, homologous to the target site recruits a nuclease – Cas9
This then cuts the dsDNA, triggering repair by either the low fidelity NHEJ pathway, or by HDR in the presence of an exogenous donor sequence.
High Efficiencies for both knockouts and knock-ins have been reported and whilst there are understandable concerns about specificity, new methodologies to address these are now being developed
The system itself is comprised of three key components
the Cas9 protein, which cuts/cleaves the DNA and
Two RNAs - a crispr RNA contains the sequence homologous to the target site and a trans-activating crisprRNA (or TracrRNA) which recruits the nuclease/crispr complex
For genome editing, the crisperRNA and TraceRNA are generally now constructed together into a single guideRNA or sgRNA
Genome editing is elicited through hybridization of the sgRNA with its matching genomic sequence, and the recruitment of the Cas9, which cleaves at the target site.
Introduction to CRISPR Cas9 technology. View in slide show after downloading for better viewing. Description is minimal, but it will be worth going through the slides that are full of pictures, if you have a minimal understanding of CRISPR.
Prepared in Oct 2015
Crispr cas9 and applications of the technologyNEHA MAHATO
The most talked about gene editing tool- CRISPR Cas9 and its applications in all the possible spheres of science and research is talked about in brief in this presentation.
Have you considered that protein over-expression or inefficient mRNA knockdown may be masking physiological effects in your assays? Increasingly scientists are moving to endogenous gene-editing to characterise the function of their genes of interest.
Dr Chris Thorne from Cambridge Biotech Horizon Discovery discusses the ground breaking gene-editing technology CRISPR. The simplicity of experimental design has led to rapid adoption of the technology across the scientific community. However, challenges remain.
This Slidedeck focuses specifically on implementing CRISPR experiments, and explore a number of key considerations crucial to maximising chances of targeting success, whether your goal is to generate a knock-out or a knock-in. Chris also takes a look at some of the alternative uses of CRISPR, including sgRNA genome wide synthetic lethality screens.
The slides aim to support those researchers either planning to or already using CRISPR gene-editing in their lab. Horizon Discovery have also recently launched a program aimed specifically at academic cell biologists to promote the adoption of CRISPR by offering FREE CRISPR Reagents for knock-out cell line generation - more information available here. http://www.horizondiscovery.com/what-we-do/discovery-toolbox/genassist-crispr--raav-genome-editing-tools
Webinar by BIS Research on Precision Oncology BiomarkersBIS Research Inc.
Precision oncology biomarkers are essential tools for tailoring cancer treatment to individual patients, as they provide insights into tumor biology and guide the selection of targeted therapies.
BIS conducted a deep intelligence webinar on the state-of-the-art technologies and emerging strategies used through the precision oncology biomarkers.
CRISPR is easily the best gene editing tool to date. For decades, scientists have been looking for a way to to perform precise changes to genetic sequences. In the past several years, researchers were able to exploit the immune systems of bacteria to edit the genome of other living cells. CRISPR is reported to have higher targeting efficiencies when compared to TALENs and Zinc Fingers. It is efficient, easy to use and cheap; making it a scalable genetic engineering tool that is highly desirable in various industry-wide applications.
Use of CRISPR-Cas9 has revolutionized targeted genome editing. However, rapid design of high-quality guide RNA (gRNA) sequences with high on-target and low off-target editing remains challenging. We implemented a machine learning algorithm to design high-quality gRNA sequences in 5 commonly used species (human, mouse, rat, zebrafish, and nematode). Our tool also designs gRNA sequences against custom targets, and can check existing gRNA designs for quality. In this webinar, we review our data illustrating this tool's performance and demonstrate its use in predicting and designing improved gRNAs for genome editing.
CRISPR/Cas9 gene editing is based on a microbial restriction system, that has been harnessed for genome targeting using only a short sequence of RNA as a guide.
The beauty of the system is that unlike protein binding based technologies such as Zinc Fingers and TALENs which require complex protein engineering, the design rules are very simple, and it is this fact that is allowing CRISPR to take genome engineering from a relatively niche persuit to the mainstream scientific community.
The principle of the system is that a short guide RNA, homologous to the target site recruits a nuclease – Cas9
This then cuts the dsDNA, triggering repair by either the low fidelity NHEJ pathway, or by HDR in the presence of an exogenous donor sequence.
High Efficiencies for both knockouts and knock-ins have been reported and whilst there are understandable concerns about specificity, new methodologies to address these are now being developed
The system itself is comprised of three key components
the Cas9 protein, which cuts/cleaves the DNA and
Two RNAs - a crispr RNA contains the sequence homologous to the target site and a trans-activating crisprRNA (or TracrRNA) which recruits the nuclease/crispr complex
For genome editing, the crisperRNA and TraceRNA are generally now constructed together into a single guideRNA or sgRNA
Genome editing is elicited through hybridization of the sgRNA with its matching genomic sequence, and the recruitment of the Cas9, which cleaves at the target site.
Introduction to CRISPR Cas9 technology. View in slide show after downloading for better viewing. Description is minimal, but it will be worth going through the slides that are full of pictures, if you have a minimal understanding of CRISPR.
Prepared in Oct 2015
Crispr cas9 and applications of the technologyNEHA MAHATO
The most talked about gene editing tool- CRISPR Cas9 and its applications in all the possible spheres of science and research is talked about in brief in this presentation.
Have you considered that protein over-expression or inefficient mRNA knockdown may be masking physiological effects in your assays? Increasingly scientists are moving to endogenous gene-editing to characterise the function of their genes of interest.
Dr Chris Thorne from Cambridge Biotech Horizon Discovery discusses the ground breaking gene-editing technology CRISPR. The simplicity of experimental design has led to rapid adoption of the technology across the scientific community. However, challenges remain.
This Slidedeck focuses specifically on implementing CRISPR experiments, and explore a number of key considerations crucial to maximising chances of targeting success, whether your goal is to generate a knock-out or a knock-in. Chris also takes a look at some of the alternative uses of CRISPR, including sgRNA genome wide synthetic lethality screens.
The slides aim to support those researchers either planning to or already using CRISPR gene-editing in their lab. Horizon Discovery have also recently launched a program aimed specifically at academic cell biologists to promote the adoption of CRISPR by offering FREE CRISPR Reagents for knock-out cell line generation - more information available here. http://www.horizondiscovery.com/what-we-do/discovery-toolbox/genassist-crispr--raav-genome-editing-tools
Webinar by BIS Research on Precision Oncology BiomarkersBIS Research Inc.
Precision oncology biomarkers are essential tools for tailoring cancer treatment to individual patients, as they provide insights into tumor biology and guide the selection of targeted therapies.
BIS conducted a deep intelligence webinar on the state-of-the-art technologies and emerging strategies used through the precision oncology biomarkers.
Ayurveda, the knowledge of life, immortalized in the form of elegant Sanskrit stanzas in the samhitas describe diagnosis and therapy of disease as well as ways to maintain positive health. Although the technical term “Pharmacovigilance” does not feature in ayurvedic texts, the spirit of pharmacovigilance is vibrant and is emphasized repeatedly in all major texts. The major goals of pharmacovigilance, namely to improve patient care and safety in relation to drug use, and thus promote rational drug use are recurrent themes of ayurvedic pharmacology (dravyaguna vigyan) and therapeutics (chikitsa).
The Conference Board of Canada - Tuesday, April 11, 2017 - Toronto, ON
"Leveraging Change Leadership: Driving Innovation Procurement Forward"
Presentació a càrrec de Antoni Gilabert, director de l'àrea de Farmàcia i del Medicament del CSC
MANAGEMENT OF ATRIOVENTRICULAR CONDUCTION BLOCK.pdfJim Jacob Roy
Cardiac conduction defects can occur due to various causes.
Atrioventricular conduction blocks ( AV blocks ) are classified into 3 types.
This document describes the acute management of AV block.
ARTIFICIAL INTELLIGENCE IN HEALTHCARE.pdfAnujkumaranit
Artificial intelligence (AI) refers to the simulation of human intelligence processes by machines, especially computer systems. It encompasses tasks such as learning, reasoning, problem-solving, perception, and language understanding. AI technologies are revolutionizing various fields, from healthcare to finance, by enabling machines to perform tasks that typically require human intelligence.
Anti ulcer drugs and their Advance pharmacology ||
Anti-ulcer drugs are medications used to prevent and treat ulcers in the stomach and upper part of the small intestine (duodenal ulcers). These ulcers are often caused by an imbalance between stomach acid and the mucosal lining, which protects the stomach lining.
||Scope: Overview of various classes of anti-ulcer drugs, their mechanisms of action, indications, side effects, and clinical considerations.
- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
Ozempic: Preoperative Management of Patients on GLP-1 Receptor Agonists Saeid Safari
Preoperative Management of Patients on GLP-1 Receptor Agonists like Ozempic and Semiglutide
ASA GUIDELINE
NYSORA Guideline
2 Case Reports of Gastric Ultrasound
micro teaching on communication m.sc nursing.pdfAnurag Sharma
Microteaching is a unique model of practice teaching. It is a viable instrument for the. desired change in the teaching behavior or the behavior potential which, in specified types of real. classroom situations, tends to facilitate the achievement of specified types of objectives.
Prix Galien International 2024 Forum ProgramLevi Shapiro
June 20, 2024, Prix Galien International and Jerusalem Ethics Forum in ROME. Detailed agenda including panels:
- ADVANCES IN CARDIOLOGY: A NEW PARADIGM IS COMING
- WOMEN’S HEALTH: FERTILITY PRESERVATION
- WHAT’S NEW IN THE TREATMENT OF INFECTIOUS,
ONCOLOGICAL AND INFLAMMATORY SKIN DISEASES?
- ARTIFICIAL INTELLIGENCE AND ETHICS
- GENE THERAPY
- BEYOND BORDERS: GLOBAL INITIATIVES FOR DEMOCRATIZING LIFE SCIENCE TECHNOLOGIES AND PROMOTING ACCESS TO HEALTHCARE
- ETHICAL CHALLENGES IN LIFE SCIENCES
- Prix Galien International Awards Ceremony
Explore natural remedies for syphilis treatment in Singapore. Discover alternative therapies, herbal remedies, and lifestyle changes that may complement conventional treatments. Learn about holistic approaches to managing syphilis symptoms and supporting overall health.
These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
Factory Supply Best Quality Pmk Oil CAS 28578–16–7 PMK Powder in Stockrebeccabio
Factory Supply Best Quality Pmk Oil CAS 28578–16–7 PMK Powder in Stock
Telegram: bmksupplier
signal: +85264872720
threema: TUD4A6YC
You can contact me on Telegram or Threema
Communicate promptly and reply
Free of customs clearance, Double Clearance 100% pass delivery to USA, Canada, Spain, Germany, Netherland, Poland, Italy, Sweden, UK, Czech Republic, Australia, Mexico, Russia, Ukraine, Kazakhstan.Door to door service
Hot Selling Organic intermediates
Factory Supply Best Quality Pmk Oil CAS 28578–16–7 PMK Powder in Stock
Committee for Advanced Therapies
1. Committee for Advanced Therapies
Dr Monica Ensini EURORDIS
Barcelona, June 21, 2013
www.eurordis.org
2. 2
1. Implementation of the Advanced Therapy Regulation
at the EMA:
1. the Committee for Advanced Therapies (CAT)
2. Regulatory centralised procedures for Advanced
Therapy Medicinal Products (ATMPs)
2. Role of Patients’ / Healthcare professionals’
organisations in the CAT
Content
Outline
Committee for Advanced Therapies
3. 3
How Policy Makers and Regulators approach
Advanced Therapy Medicinal Products (ATMP)
Other EU legislations that apply to ATMP products:
• Directive 2001/83/EC (medicinal products for human use)
• Regulation (EC) No 726/2004 (procedures for the authorisation and
supervision of medicinal products for human and veterinary use and
establishing a European Medicines Agency)
• Directive 2001/20/EC (clinical trials)
• Directive 2005/28/EC (good clinical practice, manufacture and
import)
• Directive 2003/94/EC (good manufacturing practice)
• Regulation (EC) No 1901/2006 (paediatric medicines)
• Regulation (EC) 141/2000 on Orphan Drugs
Lack of harmonisation across Europe resulted in
Regulation (EC) No 1394/2007 on ATMP
Committee for Advanced Therapies
4. 4
Regulation on Advanced Therapies
(EC) No1394/2007
Medical
Devices
Pharmaceuticals
(e.g. aspirin)
Biotech
(e.g. recombinant
insulin)
Gene TherapyCell TherapyTissue
Engineering
Advanced TherapiesScience
Legislation
Regulation on
Advanced Therapies
(EC) No1394/2007
Evaluation
CAT
expertise
CHMP
expertise
CAT
expertise
Medical
Devices Dir.
93/42/EEC
Medicinal
Products
2001/83/EC
Committee for Advanced Therapies
5. 5
• Advanced Therapy Medicinal Products (ATMP)
• Gene Therapy Products
• Somatic Cell Therapy Products
• Tissue Engineered Products
• Combined ATMP
• Principles of existing legislation on medicines apply to
advanced therapies:
• Quality, Safety and Efficacy
• Marketing authorisation
• Post-authorisation vigilance
Regulation on Advanced Therapies
Key elements
Committee for Advanced Therapies
6. 6 Committee for Advanced Therapies
What is a gene therapy product?
• Medicinal product that contains or consists of a recombinant gene
administred with a view to regulating, repairing, replacing, adding or
deleting a genetic sequence
• Type of gene therapy
• non-specific placement
• swap/repair a gene
• transcription regulation
• Vectors
• viral/non-viral/hybrid
• Transduction
• ex vivo / in vivo
• target cells
http://www.biochem.arizona.edu/classes/bioc471/pages/Lecture25/AMG9.11a.gif
7. 7 Committee for Advanced Therapies
Ex vivo Gene therapy: treatment of SCID disease
http://history.nih.gov/exhibits/genetics/sect4.htm
http://athena.bioc.uvic.ca
SCID – severe combined
immunodeficiency
8. 8 Committee for Advanced Therapies
What is a somatic cell therapy product?
• Medicinal product based on substantially manipulated cells
or tissues or of cells or tissues that are not intended to be
used for the same essential function(s) in the recipient and
the donor
• Cells / tissues from patient itself, from another human or
from animals
• Manipulated (engineered) cells / tissues (non substantial:
cutting, grinding, centrifugation, irradiation etc)
• Scope: treating, preventing or diagnosing a disease
through the pharmacological, immunological or metabolic
action of its cells or tissues
9. 9 Committee for Advanced Therapies
Example: Cancer Cell therapy
10. 10 Committee for Advanced Therapies
What is a
Tissue Engineered product?
• Tissue Engineered Products (TEP)
Contain/consist of engineered cells/tissues
Administered to human to regenerate, repair or replace a
human tissue
Examples:
Artificial skin (burn wounds)
Cartilage repair
Neo-organs
13. 13 Committee for Advanced Therapies
Gene therapy medicinal product:
- recombinant nucleic acid -> to regulating, repairing, replacing, adding or
deleting a genetic sequence
Somatic cell therapy medicinal products:
-substantially manipulated cells/tissue -> to treat, prevent or diagnose a
disease (pharmacological, immunological, metabolic action)
Tissue engineered product:
-substantially manipulated cells/tissue -> to regenerate repair or replace a
human tissue
Combined ATMP:
- medical device + cell/tissue part
ATMP: summary of definitions
14. 14 Committee for Advanced Therapies
« The Committee for Advanced Therapies (CAT) is
the committee at the European Medicines Agency
that is responsible for assessing the quality, safety
and efficacy of advanced therapy medicinal products
(ATMPs) and following scientific developments in the
field. It is a multidisciplinary committee, gathering
together some of the best available experts in
Europe.
It was established in accordance with Regulation (EC)
No 1394/2007 on ATMPs.»
http://www.ema.europa.eu
Committee for Advanced Therapies
15. 15
CAT should cover the
scientific areas relevant to
advanced therapies,
including:
- Medical devices
- Tissue engineering
- Gene therapy
- Cell therapy
- Biotechnology
- Surgery
- Pharmacovigilance
- Risk management
- Ethics.
[Recital 9 & Art.21]
The Committee for Advances Therapies (CAT):
Composition
Committee for Advanced Therapies
17. 17
►To define borderline cases
e.g. with medical device, transplant,
cosmetics.
►Incentive for applicants, not legal
requirement
►Fast procedure (max 60 days)
CLASSIFICATION
Is my product an Advanced Therapy Medicinal
Product (ATMP)?
Committee for Advanced Therapies
18. 18
► Incentive for Small to Medium-sized
Enterprises
► Assessment of early quality and non-
clinical data
► Fast procedure (90 days), confidential
► Certificate may attract investments
CERTIFICATION
Are the data generated to date sufficient?
Committee for Advanced Therapies
19. 19
► Scientific Advice can be given on ANY
scientific question
•Quality, non-clinical and clinical
► At any time point of development
•Post-marketing advice is also
available
► Broad advice, Conditional approval
and Exceptional circumstances
► Confidential
SCIENTIFIC
ADVICE
Advice on product development
For ATMPs the Scientific Advice Working Party (SAWP) consults the CAT
Committee for Advanced Therapies
20. 20
EVALUATION
DRAFT OPINION MAA
►Principles of existing legislation on medicines
apply to advanced therapies
►Centralised procedure mandatory
►CAT with specific expertise to evaluate MAAs
► Risk based approach
► Risk Management Plan and follow-up of
safety and efficacy
The product development is completed
Committee for Advanced Therapies
21. 21
CHMP Co-ordinator responsible for flow of information
between CAT & CHMP + discussion/adoption of opinion at
CHMP
ASSESSMENT
TEAM 1
ASSESSMENT
TEAM 2
CHMP Co-ordinator
(at CHMP level)
+
CAT Rapporteur
(at CAT level)
incl.
Q/S/E Experts
CHMP Co-ordinator
(at CHMP level)
+
CAT Co-Rapporteur
(at CAT level)
incl.
Q/S/E Experts
Peer review by 1 CHMP
member 1 (or more) CAT
member(s)
CAT (Co)Rapp coordinate
procedure & discussions at CAT
+ prepare draft opinions and
assessment reports
Committee for Advanced Therapies
Evaluation Procedure
22. 22 22
A medicinal product may be placed on the market
in the EU, when:
a marketing authorisation has been issued
by the EU Commission via the Centralised Procedure
(EMA)
or
it is delivered under hospital exemption, regulated by the
competent authority of an EU Member State
EU Marketing Authorisation (MA) for ATMPs
22Committee for Advanced Therapies
23. 23 23
• 1 application to EMA 1 scientific evaluation
• Scientific Committee:
CAT + adoption by the CHMP
• Maximum legal time limit
210 days evaluation (CAT Opinion + CHMP Opinion) + EU
Commission Decision
• 1 Marketing Authorisation valid for the whole EU
• 1 Trade name and 1 Labelling (all EU languages)
Summary of Product Characteristics
User Package Leaflet
Package Labelling
Centralised Procedure for ATMPs
Committee for Advanced Therapies
24. 24
1. Implementation of the Advanced Therapy Regulation at
the EMA:
1. the Committee for Advanced Therapies (CAT)
2. Regulatory centralised procedures for Advanced
Therapy Medicinal Products (ATMPs)
2. Role of Patients’ / Clinicians’ organisations in the CAT
Content
Outline
Committee for Advanced Therapies
25. 25 Committee for Advanced Therapies
Clinicians’ & Patients’ organisations in the CAT
• Second Public call for expression of interest:
closed on 1 December 2011 (2 Full Member + 2 Alternate
Member for Clinicians; 2 Full Member+2 Alternate
Member for Patients )
• Commission will review all applications and will prepare a
draft Commission Decision
• Consultation of European Parliament
• Final Commission Decision: ???
26. 26
EMA Scientific Committees and Patients’ Contribution
• Expertise: convey a combination of specific education,
training and professional experience.
• Experience: convey practical disease knowledge obtained
from direct contact with the disease
• Advocacy: act on behalf of the affected patients in defence
of their rights; provide patient-oriented public health /
healthcare policy perspective.
• Empowerment: Access to the information necessary to
participate in the decision-making processes on behalf of all
patients.
Committee for Advanced Therapies
27. 27
Role of Patients representatives in the
EU Centralized Procedures for ATMP
• Full members of the CAT
Vote on products / procedures
Stand for chair/vice-chair of CAT
Can be Rapporteur, Co-Rapporteur, Peer reviewer
Can take part of assessment team for:
– MAA for ATMP
– Re-registration of products legally on the market
– Certification of Quality/Non-Clinical data
Can be Rapporteur for scientific guidelines
Committee for Advanced Therapies
28. 28
Role of Patients representatives in the
EU Centralized Procedures for ATMP
Representing patients’ voice
Propose patients experts
Bringing points of view and perspectives on Regulatory procedure
Link outside POs useful for their specific expertise
Points of view and real life experience of concerned patients
Address issues that could concern lay people
Involvement in all the Regulatory process including issues of post-
marketing access.
Propose actions beyond the regulatory framework: e.g. proposal for a
CAT work programme addressing general issues related to ATMPs
development
Committee for Advanced Therapies
29. 29
CAT: where we are now
http://www.ema.europa.eu/docs/e
n_GB/document_library/Work_pro
gramme/2010/11/WC500099029.pd
f
Committee for Advanced Therapies
30. 30
CAT Objectives for the 2010-2015
► Facilitate development ATMP and submission of MAA:
understand trends in research and development, strengthen
dialogue with stakeholders, reinforce internal/external
cooperation
► Promote the use of available regulatory procedures and
introduce potential improvements
► Explore possibilities offered by the current regulatory
framework when applied to ATMP
► Contribute to foster innovation
► Promote access and availability to ATMP
Committee for Advanced Therapies
31. 31
ATMP Regulation implemented
Clear framework for MAA for ATMP
Proactive approach to address the needs of the sector
Early dialogue between interested parties
CONCLUSION
Committee for Advanced Therapies
32. 32 Committee for Advanced Therapies
Thank you
Thanks you for your attention!
And a very special thank to
Dr Patrick Celis EMA-CAT Scientific
Secretariat ,
Dr. Michele Lipucci di Paola (EMA-
CAT/EURORDIS) and
Dr. Fabrizia Bignami
who heavily contributed to this
presentation
