29 June 2012
SECTION 1: THE EVENT
-
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.
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.
SECTION 2: PROGRAMME
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
SECTION 3: EVALUATION
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
http://www.orpha.net/consor/cgi-bin/index.php
Also of importance were the following websites:
US National Institute of Health website: http://clinicaltrials.gov/
CONSORT: Transparent reporting of trials http://www.consort-statement.org/
BMJ Statistics Notes Series http://openwetware.org/wiki/BMJ_Statistical_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.
SECTION 4: RECOMMENDATIONS
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. http://www.rareconnect.org/en
SECTION 5: CONCLUDING REMARKS
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
Pretoria
ACRONYMS
| 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 |
| OECD |
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 |