How are medicines evaluated at the
EMA – Part I
Nathalie Bere, Patients relations co-ordinator
An agency of the European Union
What is the European Medicines Agency (EMA)
The EMA is the EU regulatory body responsible for the
scientific evaluation and supervision of medicines
developed by pharmaceutical companies for use in the
European Union (Human and Veterinary).
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European Regulatory Network
The European regulatory system for medicines is a unique model in the global
regulatory environment.
This system is based on a network that
includes all national medicines authorities
(human and veterinary) from EU Member
States and the European Economic Area,
working closely together in an integrated
manner.
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What does the EMA do
• Evaluation of marketing authorisation applications for human and
veterinary medicines submitted by pharmaceutical companies
• Provision of scientific advice on the development of medicines
• Evaluation of applications for orphan designation in EU
• Evaluation of paediatric investigation plans (or waivers)
• Coordination of European pharmacovigilance (supervision of the medicines on
the market)
• Evaluation of arbitration and referral procedures
• Provision of good quality and independent information on the medicines it
evaluates to patients and healthcare professionals
• Coordination of Member States’ inspections
3
What the EMA does not do
The European Medicines Agency does not control:
Pricing of medicines
Access to medicines
Advertising of medicines
Patents on medicines
Medical devices
Homoeopathic medicines
Food supplements
Cosmetics
Tobacco
4
Are all medicines approved via the EMA?
No. In the European Union (EU), there are two ways of getting a marketing authorisation for
a medicine:
1. Centralised authorisation procedure, via the European Commission after evaluation by
EMA, which results in a single marketing authorisation valid throughout the EU;
2. National authorisation procedures, where individual EU Member States authorise
medicines for use in their own territory through 3 possible procedures:
National authorisation
Mutual-recognition procedure (MRP)
Decentralised procedure (DCP)
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Medicines that are mandatory for evaluation at EMA
• Rare diseases
• HIV, cancer, neurodegenerative disorders, diabetes
• Auto-immune diseases, viral diseases
• All biotech products
• Gene therapy
• Monoclonal antibodies
+ Other innovative products
Medicines outside the mandatory scope can also be evaluated at EMA if they meet
certain criteria.
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The centralised procedure and the EMA
Marketing Authorisation
application
Evaluation
Authorisation in all EU MS
Invented name
Product information
Summary of Product Characteristics (SmPC) EU
Labelling languages
Package Leaflet (PL)
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EMA and its scientific committees
The EMA committees contain members nominated by the medicines regulatory authorities of the EU
Member States (the ‘national competent authorities’)
Committee for Human Committee for Veterinary
Medicinal Products Medicinal Products
(CHMP) (CVMP)
Committee for Orphan
Paediatric Committee
Medicinal Products
(PDCO)
Patient
EMA (COMP)
members
Secretariat Patient
members
Committee for Herbal Committee for
Medicinal Products Advanced Therapies
(HMPC) Pharmacovigilance Risk (CAT)
Patient
Assessment Committee members
(PRAC)
Patient
members
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Working Parties and other Groups
Working Parties CMDh
Co-ordination Group
SAWP for Mutual Recognition
QWP and Decentralised
Other working parties
Scientific advice Quality Procedures
Biosimilars
Biostatistics
Blood Products
Cardiovascular HIV / SWP
Anti-
Central Nervous System Antiviral Safety
Infectious Diseases Vaccines infectives
Oncology Working
Pharmacogenomics Diabetes
Diagnostics
Pharmacokinetics ad-hoc Endocrinol
Rheumatology/Immuno. ogy
expert
Vaccines Cardio
groups
vascular BWP
issues Psychiatry
Biologics
Oncology Neurology
QRD
Working Group on PCWP
Quality Review of HCPWP
Patients and GCP Inspectors
documents Healthcare
consumers Working group
professionals
9
Experts who work with the scientific committees
National Patients and
Agencies Consumers
EMA
Scientific Committees
Learned Healthcare
societies professionals
Academia
and
Networks
10
Patient/consumer involvement in the EMA
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Interaction with patients: the EMA journey… so far
2014 Ongoing…
Systematic
2006 • Dedicated
inclusion of real
Patients and
Patients and Healthcare life experience
2005 Consumers Professionals EMA regulatory
Framework of Working Party Department output
2003 interaction with (PCWP)created created
patient and
Working group consumer • Revised
with patients organisations Framework
2000 created
Patients join
COMP as full
members
1996
Dialogue
with HIV
1995 patients
EMA
created
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How are patients involved at EMA?
Patients representing • Management Board
patients’ organisations • EMA Scientific Committee(s)
product
• Patients’ and Consumers’ Working Party (PCWP)
related
Non-
Patients representing • EMA consultations
their organisations
• Workshops
• Scientific Advice / Protocol Assistance Procedures
Product
related
Patients as individual • Scientific Advisory / ad hoc expert Groups
experts • Scientific committee consultations
• Review of documents
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Patient involvement as individual experts in EMA activities
Pre-submission:
• Participation in scientific advice/protocol assistance procedures
Evaluation and Post-authorisation
• Participation in expert meetings (SAG and ad hoc)
• Respond to consultations on assessment of medicines from scientific committees and
working parties
• Review information on medicines: Package leaflets, EPAR summaries, safety
communications and other Agency documents for the public
14
Patient involvement along the medicine lifecycle at EMA
Documents for the Public
Public
Summaries Package Leaflets (PL) Package Leaflets (PL) Safety
of Opinion EPAR summaries (renewal) Communications
Patient Patient Patient Patient
input input input input
POST
PRE-SUBMISSION EVALUATION
AUTHORISATION
Regulatory Procedure
Paediatric
Orphan Designation/ Scientific Advice Marketing Authorisation Post Marketing
Investigation
ATMP Classification Protocol assistance Application Evaluation Authorisation
Plan
CHMP
Committee s and Working Parties
COMP/ CHMP- CAT CHMP
PDCO
CAT SAWP PRAC PRAC
Patient Patient Patient Patient COMP Patient
input input input input input
CAT SAG SAG
Patient Patient Patient
input input input
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Scientific Advice at the EMA
An agency of the European Union
Committees in human Medicines Regulatory process
POST
PRE-SUBMISSION EVALUATION
AUTHORISATION
Regulatory Procedure
Paediatric
Orphan Designation/ Scientific Advice Marketing Authorisation Post Marketing
Investigation
ATMP Classification Protocol assistance Application Evaluation Authorisation
Plan
Committee s and Working Parties
CHMP
COMP/ CAT CHMP
SAWP PDCO
CAT PRAC PRAC
COMP
CAT/
SAG SAG
COMP/
PDCO
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Scientific Advice
• Pharmaceutical companies can request scientific advice from the EMA
regarding the development of a medicine.
• Aimed at ensuring the most appropriate studies are conducted, avoiding major
objections related to the study design during evaluation
• The Scientific Advice Working Party (SAWP) and the Committee for Medicinal
Products for Human Use (CHMP) provide scientific advice by answering specific
questions posed by the companies.
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Types of questions
Scientific Advice can be provided on questions ranging from:
• Quality – manufacture of medicines
• Non-clinical – animal studies – interpretation and extrapolation of results
Clinical – discussion of study population, endpoints, feasibility of trial
• Regulatory – including statistics
Significant benefit – for orphan medicines (where applicable)
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The role of patients and patient representatives
Patient representatives are invited to participate in EMA scientific advice
procedures:
• Either face to face meeting or via written comments
• Share their ‘real-life’ perspective and experience with the SAWP and the
pharmaceutical company, in relation to a particular medicine in their disease
area.
• Provide comments on the development proposals from the company (e.g.
endpoints, population, feasibility etc)
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Scientific Advisory/ad hoc expert Group
meetings
An agency of the European Union
Committees in human Medicines Regulatory process
POST
PRE-SUBMISSION EVALUATION
AUTHORISATION
Regulatory Procedure
Paediatric
Orphan Designation/ Scientific Advice Marketing Authorisation Post Marketing
Investigation
ATMP Classification Protocol assistance Application Evaluation Authorisation
Plan
Committee s and Working Parties
CHMP
COMP/ CAT CHMP
SAWP PDCO
CAT PRAC PRAC
COMP
CAT/
SAG SAG
COMP/
PDCO
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Joint List of Outstanding
Start Assessment List of Commission
Assessment Issues/ Oral Opinion
Report Questions Decision
Day 1 Report explanation Day 210
Day 80 Day 120 Day 277
Day 150 Day 180
Evaluation of Assessment on
benefit/risk need for post
safety/efficacy
studies
Assessment of Product
Assessment Information Preparation of RMP
of Risk summary
Management
Plan (RMP)
Additional information:
•Brochure – Applying for marketing authorisation
23 •CHMP overview
Type of Approvals
Standard:
Comprehensive data
Conditional Approval:
• Comprehensive data not
available; to be provided after
approval
approval • Must fulfil scope (orphan drugs,
emergency threats, serious and
life-threatening diseases)
Approval valid for 1 year,
renewable
Exceptional Circumstances:
• Comprehensive data not available and cannot be provided
• Must meet criteria (rarity, medical ethics, state of scientific knowledge)
For these hyperlinks – please refer to question 56
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Scientific Advisory/Ad hoc expert Groups
• The CHMP or the Pharmacovigilance and Risk Assessment Committee (PRAC) can convene
a SAG during the evaluation of a specific medicine when they encounter specific questions
that are best answered by experts in the field, including patients
• SAGs exist for specific therapeutic areas and when an issue arises for which there is no
SAG, an ad hoc expert group is organised
• Two patients, with experience of the disease/condition, are invited to participate in every
SAG / ad hoc expert group meeting
• Patients contribute by providing input to the discussions on the benefits and risks, from
their perspective in relation to the questions that the CHMP is asking
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Contact
Nathalie Bere
Patient relations
Stakeholder and Communication Division
[email protected]
www.ema.europa.eu
[email protected]
European Medicines Agency
30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom
Telephone +44 (0)20 3660 8452 Facsimile +44 (0)20 3660 5550
Send a question via our website www.ema.europa.eu/contact
Follow us on @EMA_News
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