june 2-6,2014 programme capacity building programme for ... · eurordis summer school for patient...

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



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




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)




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)




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




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


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


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

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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)


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




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.


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



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




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


mailto:[email protected]

june 2-6,2014 programme capacity building programme for ... · eurordis summer school for patient...

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