1. Expert Patients and Researchers EURORDIS Summer School “ExPRESS Yourself!”
A capacity building programme
for patient representatives
& researchers on information and access to orphan, paediatric, advanced therapies and health technology assessment.
Nancy Hamilton

2. Overview of the programme
Medical research & development.
Ethics in medical research & development
Statistics in medical research
Regulatory Procedures.
European Medicines Agency.
Pharmacovigilance and Benefit Risk.
Market Access and Health Technology Assessment.

3. EVIDENCE-BASED MEDICINE
Care of individual patients is based on evidence
From medical research
From medical expertise and experience
Medical research “gold standard”: randomised, controlled trial (RCT)
Methodological principles

4. Studies should be conducted ”double-blind” whenever possible
SHORTCOMINGS?
Randomisation does not guarantee that the study setting is free from bias, particularly if
The participant, the investigator or both know what treatment the individual will receive (open-label study)
Studies should be conducted ”double-blind” whenever possible
Methodological principles

5. TO CONCLUDE
Randomised, controlled, blinded studies provide best scientific evidence of benefit and risk
Not always possible
Alternative designs to be agreed with authorities in advance
Methodological principles

6. Medical research involving human beings: an attempt at a definition
‘The primary purpose of medical research involving human subjects is to understand the causes, development and effects of diseases and improve preventive, diagnostic and therapeutic interventions (methods, procedures and treatments)…’
(Declaration of Helsinki, General Principles art. 6)
Ethical Aspects of Medical Research Involving Human Beings

8. Which benefits for which risks?
Safety?
Efficacy?
Quality?
François Houÿez
Eurordis Summer School 2017, Barcelona
Which benefits for which risks?

9. A closer look: Autonomy Informed consent on the basis of full information
The right to refuse to be included without
repercussions on subject treatment
The right to withdraw at any time
(without giving an explanation)
without repercussions on participant treatment
Identity of the investigator and information
on how to reach her/him
Opportunity to ask questions and consult
an independent physician
The opportunity to consult family, acquaintances
or one’s own General Practitioner
Ethical Aspects of Medical Research Involving Human Beings

10. 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)

11. What is the EMA doing? 27 February 2018
Facilitate development and access to medicines
Evaluate
marketing authorisation applications
Protect human and animal health
Provide information on medicines to healthcare professionals and patients
Monitor the safety of medicines throughout their life cycle
Presentation title (to edit, click Insert > Header & Footer)

12. Centralised Procedure Individual member state(s)
27 February 2018
The European System
In Europe all medicines must have a marketing authorisation before they can be put on the market
There are two ways of obtaining an authorisation:
Centralised authorisation procedure
National marketing authorisation procedures
National
procedures
Decentralised
Procedure
Mutual
Recognition
Centralised Procedure
EMA
(All member states)
Individual member state(s) 12 12

13. + working parties and scientific advisory groups
How is EMA organised?
Management
Board
Executive
Director
EMA staff
CHMP
Committee for
Human
Medicines
COMP
Committee for
Orphan
Medicines
HMPC
Committee for
Herbal
Medicines
PDCO
Paediatric
Committee
CAT
Committee for
Advanced
Therapies
PRAC
Pharmacovigilance
Risk Assessment
Committee
+ working parties and scientific advisory groups
EU institutions
~4000 European experts
National Agencies
Healthcare professionals
Patients
Academia
Learned societies

14. List of Outstanding Issues/ Oral explanation
Evaluation overview - CHMP
Joint Assessment
Report
Day 150
List of Outstanding Issues/ Oral explanation
Day 180
Start
Day 1
Assessment
Report
Day 80
List of Questions
Day 120
Commission Decision
Day 277
Opinion
Day 210

15. How do we monitor the safety of medicines already on the market?
27 February 2018
How do we monitor the safety of medicines already on the market?
Final decision from the EC
Communication to the network
Reports from patients and healthcare professionals
CHMP
Clinical studies
CAPs + NAPs
EMA/PRAC assessment
Medical literature
PRAC recommendation
Regulatory bodies outside the EU
NAPS only
Final decision from CMDh
Patient registries
Various/potential data inputs received that might lead to safety concerns
Presentation title (to edit, click Insert > Header & Footer)

16. Patient contribution
Patient involvement in MoCA is essential to bring the patients’ voice to the table as legitimate experts on:
The disease they are suffering from
The disease’s impact on their daily lives
The solutions offered by available medicines
The unmet needs that new treatments should aim to fill
The impact of the therapy in real-life
Patient-relevant outcome measures
How patients will be affected if the medicine is only accessible in some countries and not others

17. Three categories of patient participation:
Patients representing the patient community
Patients representing their organisation
Patients as individual experts 1 2 3

18. 1. Patients representing the patient community
Members of :
EMA Management Board (MB)
Committee for Orphan medicines (COMP)
Committee for paediatric medicines (PDCO)
Committee for advanced therapies (CAT)
Pharmacovigilance Risk Assessment Committee (PRAC)

19. Orphan drug legislation
The main objective of the Orphan Regulation is to ensure that patients suffering from rare conditions have the same quality of treatment as any other patient in the EU
Incentives:
marketing exclusivity in the EU for 10 years after approval
protocol assistance
access to the Centralised Procedure for Marketing Authorisation