EURORDIS SUMMER SCHOOLFOR PATIENT ADVOCATES
IN CLINICAL TRIALS AND DRUG DEVELOPMENT
EURORDISRare Diseases Europe
ProgrammeJune 2-6, 2014
A capacity building programme for patient representativesinvolved in the development, information and access to orphan,
paediatric, advanced therapies,and health technology assessment
Co-funded by:
Co-funded by AFMTELTHONthe Health Programme INNOVER POUR CUERIRof the European Union
Developed with the support of:
0 PLATAFORMA I I I I I Fundació Doctor RobertUFB MALALTIES MINORITARIES UAB
0 !nsermEUROI’EAN ML1)ICIN[S AGENCY InstItt natonuI
U UN MItCI.S AL II delasantêetdetech.rchemidkal.
Organisers:Dr. Maria Mavris Nancy Hamilton Platforma Malaties MinoritàriesEURORDIS EURORDIS lolanda Arbiol
Final Report - CHAFEA Operating Grant Nr: 20133304 - EURORDIS FY2014 B.2.c.2. Summer School 2014_Annex 1. ESS Programme20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
EURORDIS SUMMER SCHOOLFOR PATIENT ADVOCATES
IN CLINICAL TRIALS AND DRUG DEVELOPMENT
EURORDISRare Diseases Europe
June 2-6, 2014BARCELONA, SPAIN
Day3June 5th
Regulatorycommittees andworkig parties at theEurobean Medicines
Agency
Welcome Dinner2 June
Dayl 1 Day2June 4thJune 3rd
Life-cycle of DrugDevelopment
Principles of clinical trials
Ethics
Post-marketing phases
Health TechnologyAssessment
Day4June 6th
Regulatory committeescont...
EURORDIS activities
Final Report - CHAFEA Operating Grant Nr: 20133304 - EURORDIS FY2014 B.2.c.2. Summer School 2014_Annex 1. ESS Programme20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
EURORDIS SUMMER SCHOOLFOR PATIENT ADVOCATES
IN CLINICAL TRIALS AND DRUG DEVELOPMENT
PROGRAMME
Tuesday June 3, 2014
15:00-16:30
Dayl
EURORDIS Welcome Address and Introduction08:45-09:00
Fundacio Dr. Robert to Summer School
09:00-10:00 Small group discussions using session 1 material provided
Clinical Research
. Need for evidence-based medicine
• . Life cycle of drug development from pre-clinical10:00-11:00 Dr. Markku Toivonen
(specificity of orphan medicinal products)
. Diagram demonstrating stages of drug
development.
11:00-11:30 Coffee break
11:30-12:30 Small group discussions using session 2 material provided
Methodology principle in clinical trials
. The ‘Gold Standards’
• > Controlled12:30-13:30 Dr. Markku Toivonen
> Blind
Randomised
> Small populations
Lunch
(Participants present themselves)
Methodological principles
. Statistical significance
.. Clinical significance
rof. John Norrie• p value
. Statistical power
. Statistical risks
1St Focus Group on ethical and social issues on data sharing:
Led by Pauline McCormack and Anna Kole
(Interested participants will have to register separately if they would like to
attend. This session will be held at the Hotel Alimara)
Final Report - CHAFEA Operating Grant Nr: 20133304 - EURORDIS FY2014 B.2.c.2. Summer School 2014_Annex 1. ESS Programme20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
EURORDIS SUMMER SCHOOLFOR PATIENT ADVOCATES
IN CLINICAL TRIALS AND DRUG DEVELOPMENT
PROGRAMME
Wednesday June4, 2014 Day2
09:00-10:00 Group discussions using session 3 material provided
Ethical aspects
. Therapeutic v Experimental10:00-11:00 ds Eric Koster MA
situation
. Consent for participation
11:00-11:30 Mr. Rob Camp Ethical Aspects from a US perspective
11:30-11:45 Coffee Break
11:45-12:45 Group discussions using session 4 material provided
Regulatory procedures
. Importance of Post-Marketing
phases
. Compassionate use
12:45-13:45 Dr. Patrick Salmon • Accelerated review
. Conditional Approval
. Marketing Authorisation under
exceptional circumstances
• Risk management plans
13:45-15:00 Lunch
,::i’
15:00-16:30 . Edmond Jessop • Introductory HTA workshopf
I
2nd Focus Group on ethical and social issues on data sharing:
Led by Pauline McCormack and Anna Kole19.00
(Interested participants will have to register separately if they would like to
attend. This session will be held at the Hotel Alimara)
Final Report - CHAFEA Operating Grant Nr: 20133304 - EURORDIS FY2014 B.2.c.2. Summer School 2014_Annex 1. ESS Programme20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
EURORDIS SUMMER SCHOOLFOR PATIENT ADVOCATES
IN CLINICAL TRIALS AND DRUG DEVELOPMENT
PROGRAMME
Thursday June 5, 2014 Day3
• General Introduction to the European Medicines09:00-09:30 Ms. Nathalie Bere
Agency
09:30-10:15 Dr. Jordi Llinares Garcia Committee for Orphan Medicinal Products (COMP)
10:15-11:15 Mini-COMP session
11:15-11:30 Coffee Break
Prof. Josep Torrent i11:30-12:30 Scientific Advice Working Party (SAWP)
Farnell
. Committee for Medicinal Products for Human Use12:30 - 13:15 Dr. Patrick Salmon
(CHMP)
13:15-14:45 Lunch
14:45-16:30 Training on Review of Product Information -
(Includes S Ms. Nathalie Bereworkshop
minute break)
Tour of the Cosmo Caixa Museum
Final Report - CHAFEA Operating Grant Nr: 20133304 - EURORDIS FY2014 B.2.c.2. Summer School 2014_Annex 1. ESS Programme20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
EURORDIS SUMMER SCHOOLFOR PATIENT ADVOCATES
IN CLINICAL TRIALS AND DRUG DEVELOPMENT
PROGRAMME
Friday June62014 Day4
Dr. Fernando de Andres09:00-09:30 Paediatric Committee (PDCO)
Trelles
09:30-10:30 Mini-PDCO session
10:30-11:00 Dr. Michele Lipucci di Paola Committee for Advanced Therapies (CAT)
11:00-11:15 Coffee break
11:15-12:00 Ms. use Murphy Patients’ and Consumers’ Working Party (PCWP)
Pharmaceutical Risk Assessment Committee12:00-12:30 Mr. Albert van der Zeijden
(PRAC)
12:30-13:00 Dr. Christine Kubiak Presentation of ECRIN project
13:00-14:00 Lunch
• •. Pharmacovigilence: the Role of Patient14:00-14:25 Mr. François Houyez
Associations other than the PRAC
-‘ .
EUPATI: European Patients Academy for14:25-14:45 Fr. Rob Camp
, Therapeutic Innovation
14:45-15:30 Open discussion and Closing of Summer School
EU RORD ISRare Diseases Europe
Final Report - CHAFEA Operating Grant Nr: 20133304 - EURORDIS FY2014 B.2.c.2. Summer School 2014_Annex 1. ESS Programme20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
A capacity building programme for patient representatives involved in the development, information and access to
orphan, paediatric, advanced therapies, and health technology assessment
Developed with the support of:
www.eurordis.org
EURORDIS Summer School for Patients’ Advocates in Clinical Trials and Drug Development
Barcelona, Spain - June 2-6, 2014
For more information, contact: [email protected]
EURORDIS SUMMER SCHOOL
20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
EURORDIS SUMMER SCHOOL
FOR PATIENT ADVOCATES
IN CLINICAL TRIALS AND DRUG DEVELOPMENT
Day 1
June 3
Life cycle of Drug Development
Principles of clinical trials
Day 2
June 4
Ethics Post-marketing phases
HTA
Day 3
June 5
Regulatory committees and working parties at the EMA
Day 4
June 6
Regulatory committees cont…EURORDIS
activities
www.eurordis.org
JUNE 2-6, 2014 BARCELONA, SPAIN
EURORDIS Summer School for Patient Advocates
in Clinical Trials and Drug Development
Welcome Dinner
June 2nd, 2014
at 20:00
20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
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20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
Application Form EURORDIS Summer School Barcelona, June 2 - 6, 2014
Training Rare Disease Patient Advocates in Clinical Trials and Drug Development
(Please complete all sections: only completely filled forms will be considered)
1. Your contact details
First name Last name
E-mail Telephone
Address Postal code
City Country
2. Contact details in case of emergency
First name Last name
Telephone Mobile phone
Address Postal code
City Country
3. Your organisation
Name:
Disease(s) represented: Type of organisation (please tick only one):
Governmental Non-governmental/not-for-profit Educational/research institution Transnational/Intergovernmental Informal Other (please specify):
E-mail Website
Telephone Fax
Address Postal code
City Country
4. Your role in the patient organisation Are you? Please tick all that apply
Patient Staff
Family of patient Volunteer
How long have you been active in the organisation? What are your roles or activities? (10 lines max)
20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
Do you represent your organisation in any European Committee/Task Force/ Working Group? If yes, please specify
What is your professional background? (10 lines max.)
5. English language skills Please rate your English language skills
Reading Writing Speaking Overall
Native/Advanced Good Intermediate Basic None
Native/Advanced Good Intermediate Basic None
Native/Advanced Good Intermediate Basic None
Native/Advanced Good Intermediate Basic None
6. Your experience/knowledge (5 lines max.) Please describe any past and current involvement in clinical trials and drug development: 7. Drug Development (10 lines max.) Please describe your past or potential drug development collaborations with research groups, pharmaceutical companies or actions for the development of drugs in your disease. 8. Expectations (10 lines max.) Please describe your expectations related to this summer school and how you think this training will help you in your activities.
20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme
9. Please describe your experience in:
Design and objectives of clinical trials and the roles of all
stakeholder
Patients’ roles & responsibilities in innovative medicines development
Interaction with stakeholders (regulators, industry, etc.) in drug
development processes Advanced (5+ years of
experience) Good (3-5 years of experience) Intermediate (1-3 years of
experience) Basic ( under 1 year of
experience) None
Advanced (5+ years of experience) Good (3-5 years of experience) Intermediate (1-3 years of
experience) Basic ( under 1 year of experience) None
Advanced (5+ years of experience) Good (3-5 years of experience) Intermediate (1-3 years of
experience) Basic ( under 1 year of experience) None
Medicines development process from pre-clinical research to
approval
Drug safety and risk/benefit assessment of medicines
Pharmaco-economics, health economics and health technology assessment
Advanced (5+ years of experience)
Good (3-5 of years of experience)
Intermediate (1-3 years of experience)
Basic ( under 1 year of experience)
None
Advanced (5+ years of experience) Good (3-5 of years of experience) Intermediate (1-3 years of
experience) Basic ( under 1 year of experience) None
Advanced (5+ years of experience) Good (3-5 years of experience) Intermediate (1-3 years of
experience) Basic ( under 1 year of experience) None
10. Commitment Please evaluate your level of commitment to potentially be involved in clinical trial and drug development policies and procedures, to share knowledge and exchange experience and to represent rare disease patients at the national and European level. If I am selected to attend the EURORDIS Summer School 2014: I agree to attend the full 4-day programme I agree to be included in EURORDIS list of potential volunteers so as to act as a rare disease patient
representative for activities related to clinical trials and drug development. As part of the EURORDIS list, I agree to: Share my knowledge Share my experience Be appointed as “patient expert” in particular for meetings at the EMA such as Protocol Assistance for
my disease or review of European Public Assessment Reports To review EURORDIS’ position papers, contributions and statements for advocacy purposes in the
clinical trial and drug development area Participate in other conferences and workshops I am interested in attending future capacity building sessions and seminars in orphan drug
development organised by EURORDIS or third parties in conjunction with EURORDIS. Signature Date Please fill out this form and send it back to [email protected] or fax +33 1 56 53 52 15
20133304_D08-00_OTH_EN_PS.pdf B.2.c.2. Summer School 2014_Annex 1. ESS Programme