REPORT: EURORDIS Summer School for Patient Advocates in Clinical Trials and Drug Development

29 June 2012


  • Introduction

A capacity building programme for patient representatives involved at the European level in the development, approval, information and access to orphan drugs, paediatric drugs and advanced therapies.

There were 37 attendees at the training from 17 countries, representing 28 rare diseases. Ms Joy Rosario. Chair of PiNSA and Board member of IPOPI attended from South Africa.Spain

The presenters were:

  • Dr Fernando de Andres-Trellis
  • Dr Juan Garcia Burgos
  • Mr Rob Camp
  • Mr Francois Houyez
  • Ds E Koster MA
  • Dr Christine Kubiak
  • Ms Julia Saperia
  • Dr Markku Toivonen
  • Professor Josep Torrent-Farnell

The Summer School started with a welcome dinner and was then followed by four days of training starting at 09:00 each day and ending at 16:00. A cocktail party was organised for the second evening and a tour of the Science Museum for the third.

Accommodation, local transport and food were provided by EURORDIS and flights and airport taxi by IPOPI. Preparation for the Summer School consisted of e-mailed documentation and introductions. The organisation of the event was largely managed by Maria Mavris of EURORDIS. Each person was presented with a very comprehensive file consisting of the contact information, programme, biographies, presentations and glossary of terms. Also support material for workshops.


Day 1

Welcome by EURORDIS

Clinical research: life cycle of drug development and phases of clinical trials

Methodology principles of clinical trials: how to conduct a clinical trial
Introductory statistics for medical research

Day 2

Ethical aspects of medical research involving human subjects

Regulatory procedures

EURORDIS training resources

Day 3

Introduction to the European Medicines Agency

Update on COMP activities (Committee for Orphan Medicinal Products)

Committee for Human Medicinal products (CHMP)

PWCP: Patients and Consumers Working Party (IPOPI is a member)

Workshop on the review of product information by patients in preparation for potential future consultation

Regulatory committees and working parties at the European Medicines Agency

Day 4

PDCO Paediatric Committee

CAT Committee for Advanced Therapies

SAWP: Scientific Advice Working Party and Protocol Assistance

ECRIN (European Clinical Research Infrastructure Network) and support to multinational clinical research

EURORDIS charter for clinical trials in rare diseases


The programme was well balanced taking delegates new to the concept of Clinical Trials through from the known to the unknown. It really helped having a glossary of terms to study during the course and to make sense of at the end. Acronyms needed to be deciphered and I have included a list of these at the end.

The content on regulatory committees was a challenge especially as there is a lot of cross-over amongst them. The most interesting sections for me personally were on understanding the phases of clinical trials, exploring the ethics, the use of statistics in medical research, evaluating the product information and of course the committee for advance therapies, gene therapy particularly.

The website of EURORDIS and also the EMA are excellent resources as well as ORPHANET

Also of importance were the following websites:

US National Institute of Health website:

CONSORT: Transparent reporting of trials

BMJ Statistics Notes Series

The issue of pharmovigilance was interesting and the statement that clinical trials are really experiments, thus the importance of informed consent.

One was encouraged by the stringent processes in place in Europe that ensure that patients not only receive optimum care but they are also heard, their opinion is seen as an important part of the regulatory process.


Recommendation 1: Although the Summer School is for European delegates and I attended because of my position on the IPOPI Board, it was an invaluable experience especially for someone from another country. It is recommended that other national member organisations under the umbrella of IPOPI can benefit through inviting speakers on the topic of clinical trials.

Recommendation 2: That IPOPI/PINSA joins the rare diseases social media platforms.


A really important aspect of attending the Summer School was meeting the other attendees and also the presenters. I have come away with new friends who share common issues and have also benefited greatly from advice on South Africa. I thank EURODIS, IPOPI and PiNSA for allowing me the opportunity to attend.

Joy Rosario

PiNSA: Chair




ACTG AIDS Clinical Trials Group
ADRs Adverse Drug Reactions
ATAC Aids Treatment Activists Coalition
ATMP Advanced Therapy Medicinal Products
BMJ British Medical Journal
BPWP Blood Products Working Party
BWP Biologics Working Party
CAT Committee for Advanced Therapies
CHMP Committee for Human Medicinal Products
COMP Committee for Orphan Medicinal Products
CPMP Committee for Proprietary Medicinal Products
CTMP Comprehensive Treatment and Management Plan
DG SANCO Directorate General for Health and Consumers (santé et consommateurs)
DIA Drug Information Association
EATG European AIDS Treatment Group
EBM Evidence-Based Medicine
ECRIN European Clinical Research Infrastructures Network
EFPIA European Federation of Pharmaceutical Industry Associations
EMA European Medicines Agency
EMEA European Medicines Agency
EPAR European Public Assessment Report
EU European Union
EU-RMP EU Risk Management Plan
EURORDIS Rare Diseases Europe
FDA Food and Drug Administration
FDAAA Food and Drug Administration Amendments Act
GMP Good Manufacturing Practice
GTWP Biostatistics Working Party
HCPWG Healthcare Professionals Working group
HMPC Committee for Herbal Medicinal Products
HTA Health Technology Assessment
ICH International Conference on Harmonisation
ITF Innovation Task Force
MAA Marketing Authorisation Application
NBAC National Bioethics Advisory Commission
NEJM New England Journal of Medicine
NORD National Organization for Rare Disorders

Organisation for Economic Co-operation and Development

PA Protocol Assessment
PARACELSIUS Policy Action and Information for Rare Diseases in Europe
PARD Community Action for Rare Diseases
PCO Patient and Consumer Organisations (IPOPI involved)
PCWP Patients and Consumers Working Party
PDCO Paediatric Committee
PDUFA Prescription Drug User Fee Act (1992 USA)
PhVWP Pharmacovigilance Working Party
POLKA Patient consensus on Preferred Policy Scenarii for Rare Diseases
PRAC Pharmovigilance Risk Assessment Committee
PSUR Periodic Safety Update Report
QRD Working Group on Quality Review of Documents
QSE Quality, Safety, Efficacy
QWP Quality Working Party
RAPSODY Rare Disease Patient Solidarity
RMP Risk Management Plan
SAWG Scientific Advice Working Group
SAWP Scientific Advice Working Party
SciARG Scientific Advice Review Group
SME Small Medium Enterprise
SmPC Summary Product Characteristics
SWP Safety Working Party
TAG Treatment Action group
TOPRA The Organisation for Professionals in Regulatory Affairs
VWP Vaccines Working Party