6th European Patients’ Rights Day
The EMA Geriatric Medicines
Strategy
and the empowered aging patient
Francesca Cerreta
EMA (European Medicines Agency)
1
Marketing Authorisation - Key Principles
• The EU is a Single Market for pharmaceuticals approx. 0.5 billion people.
• In order to sell a medicinal product in the EU, a
company needs a Marketing Authorisation
• There are a number of ways ( ‘Procedures’ ) for a
company to obtain a marketing authorisation.
• The main scientific principle used in the evaluation
of medicines is the benefit/risk balance, based
mainly on quality, efficacy and safety aspects
Centralised Procedure
1 application
1 evaluation
1 authorisation for all EU
1 product information
(SPC, Labelling, PL)
All EU languages
Which medicines are evaluated at the EMA?
• Rare diseases (i.e “orphan”)
• HIV, cancer, neurodegenerative
disorders, diabetes
• Auto-immune diseases, viral diseases
• All biotechnology products
• Gene therapy
• Monoclonal antibodies
+ Other innovative products
The importance of patient input for the work of
the European Medicines Agency
Framework of interaction: principle of
patient empowerment
• Patient involvement /patient information/
patient safety
• Role of patients/consumers’ organisations
as multipliers of the interaction (promoting
patient safety)
Selection criteria for involvement of patients’
organisations
• Legitimacy
• Mission/objectives
• Activities
• Representativity
• Structure
• Accountability and consultation modalities
• Transparency
Patient involvement in the Agency’s activities:
• Patients and Consumers Working Party (PCWP)
• Full members of Management Board, COMP (orphan),
PDCO (paediatric), CAT (advanced therapies)
• Review of product information:
-EPAR summaries, package leaflets, safety information Q&A
• CHMP (Ad-hoc collaboration):
– Input on assessment of products
– Experts in scientific advice/protocol assistance
– Input in guideline preparation
– Observers in Pharmacovigilance Working Party (pilot)
• Regular participation in Agency’s workshops and
conferences (e.g. Oncology QoL)
EMA Vision for a geriatric strategy:
TWO PRINCIPLES
Medicines used by geriatric
patients must be of high
quality, and appropriately
researched and evaluated..
for use in this
population.
Improve the availability
of information on the
use of medicines for
older people
Evidence based
medicine
Informed
prescription
What about the benefit / risk balance in the
older population?
•Is the benefit/risk demonstrated for the
population that will use the product?
•Which studies have been carried out? Are they in
line with current guidelines?
•Can relevant information be found in the EMA
approval documents?
•What would prescribers, patients and HTA bodies
like to know?
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EMA Geriatric Medicines Strategy - Key points
1. “..ensuring that the development and evaluation of
new medicines takes into account specific safety and
efficacy aspects related to aging, in accordance with
current guidelines, particularly ICH E7”
2. “..identifying gaps in regulatory and scientific
knowledge and taking appropriate measures to
tackle them”
3. “..consideration for the need of specific
pharmacovigilance activities”
4. “..fostering and utilising a relevant experts’ pool to
address specific issues as requested by the CHMP.”
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General Considerations
from EMA geriatric workshop 22-23 March
•Older people in many cases constitute the main
users of a drug, not a special population.
•Older adults are underrepresented in clinical trials
(relative to disease prevalence) but the situation
seems to be improving.
•Following ICH E7 Q&A, a representative number
of patients should be studied pre-authorisation.
•Data should be presented for the entire age
spectrum
•Population PK should be performed and will help
informed prescription
Changes to the CHMP Assessment Report
•Effective from October 2011
•Aim is to focus the attention of reviewer on
geriatric data:
– Amount
– Context
– Missing information
•Specific safety and efficacy sections of AR
Other Aspects
Endpoints - Depending on frailty/disability the
desirable outcome and treatment decisions might
differ with age
Clinical trial recruitment - Make use of existing
patients’ networks + CT outcome measures should
be relevant to old people
Product information-Channels of information are
important- specific measures are needed
particularly as the older group is non-homogeneous
Better focus on Package leaflet: explain how to take
medicine/increase compliance/PK and PD
changes/concomitant medication
Pharmacovigilance: Patient reporting, signal
detection; post-authorisation studies.
Appropriate formulations?
Process
Development
Formulation
Development
Drug
?
Weighing
Manufacturing
Drug
application
High quality
pharmaceutical
product
Added value of involving patients in EMA (1)
• Bring a unique and critical input based on their real-life experience
of the disease and its current therapeutic environment.
• Increase transparency and build confidence and trust in the
regulatory process.
• Representing patients interests and providing a “patient
perspective” view, on behalf of those directly affected by regulatory
decisions
• Bringing experience of the disease and/or identifying patients with
experience of the disease when necessary
Added value of involving patients in EMA(2)
• Actively contributing to patient information and communication
related to medicines. Ensure that patients and patient’s
organisations can access to useful and understandable information.
• Disseminating committees’ outcomes when they become public;
passing on information to other patients and patients’
organisations.
• Advising and supporting regulators in its dialogue with industry and
other stakeholders when identifying areas of medical need for
target research.
Thank you!
Questions?
francesca.cerreta@ema.europa.eu