Summer School 2011: from Strength to Strength
43 patients from 19 countries, representing 24 rare diseases met this summer for what is becoming an institution at EURORDIS: the Summer School for Patient Advocates in Clinical Trials and Drug Development. The event took place on June 13-16 at the Science Museum Cosmo Caixa in Barcelona. In 2011, more applications were received than in previous years, demonstrating the growing awareness of the need for training and the reputation of this course.“Overall, participants felt empowered by the knowledge they had gained and while they each entered the course with different levels of experience and expectations, they all left with a greater understanding of the drug development process and a greater sense of their role and their ability to implement changes,” states Dr Maria Mavris, Drug Development Programme Manager at EURORDIS and Summer School Coordinator. “As with each Summer School, friendships were formed, collaborations were planned and the sense of community was strengthened.”
The first two days covered the background aspects of drug development and clinical trials. Participants were introduced to the life cycle of drug development from the clinical stage to market authorisation. They became familiar with the ‘gold standards’ in clinical research and had terms explained such as blinded, controlled and randomisation.
Drug development in non-standard situations such as in the case of orphan or paediatric drugs and associated complications were described. At regular intervals students broke out into smaller groups to discuss particular documents in relation to the presentations, including orphan drug cases studies, an informed consent form, a patient information leaflet. They were also asked to examine a protocol synopsis for the drug case they were given. Particular emphasis was placed on participants’ interactions and sharing of their own knowledge and experiences. Patient representatives are regularly consulted by the European Medicine Agency (EMA) via Protocol Assistance (Scientific Advice) to give input on particular aspects of drug development for their specific rare disease, such as study endpoints and objectives.
Ethical aspects of clinical trials were also described, in particular learning to differentiate clinical versus experimental situations and better understanding the implications of informed consent.
The first half of the programme concluded with an informative session on regulatory procedures. Issues such as conditional approval, exceptional circumstances, compassionate use and accelerated review were described in detail.
The third and fourth day were devoted to the regulatory aspects of drug development and the work of the EMA. The different scientific committees of the EMA were described, with particular focus on how patient experts can be involved in such activities.
This was followed by EMA training on review of product information. The EMA training consisted of the evaluation of summaries of European Public Assessment Reports (EPARs) for medicinal products that have received marketing authorisation. Patient organisations are needed to review these public documents. The Summer School session was a summarised version of the one-day training conducted by EMA. Participants nominated as patient experts for the EMA database receive this type of training.
Following the conclusion of the EMA committee descriptions, the participants were introduced to the EURORDIS Recommendations for Good Collaboration between Patient Organisations and Sponsors of Rare Disease Clinical Trials. Having been introduced to the basic concepts of clinical trials as well as understanding how their input is necessary and required, the Clinical Trials Charter is a document that enables patient organisations to clarify their collaboration with clinical trial sponsors regarding their input and to be as transparent as possible concerning funding and access to trial results.
A brief introduction to new EMA website and the new online EU Clinical Trials Register were given, followed by a guided tour of the on-line learning tool, which is being developed by EURORDIS and INSERM to explore areas such as research, ethics, methodology and statistics, for the purpose of either continued training for Summer School participants or independent learning for member patient organisations.
“The Summer School enables the building of rare disease patient advocates’ capacities, to empower them to take a more proactive role in these policy issues as well as to work with regulatory agencies, HTA agencies, industry and academia,” explains EURORDIS Chief Executive Officer Yann Le Cam. “It is also a tool to create networks of individuals through EURORDIS who have a lot in common to share and it is a way of identifying the future leaders of the rare disease patient community.”
Since the inception of the course in 2008, 116 rare disease patient advocates from 27 countries representing 45 rare diseases have participated in the Summer School. This growing body of alumni has been trained to play a role in the drug development process and can spread its knowledge amongst patient organisations.
To view the programme and access the video presentations of all sessions (clinical trials, Health Technology Assessment and European Medicines Agency) go to the Training section of the EURORDIS website. These videos will enable Summer School participants to revise and refresh their knowledge as well as providing a learning opportunity for all patient representatives to listen to a subject of their choice.Hear about the experiences of participants and trainers and discover the benefits of the EURORDIS Summer School through a series of interviews.
This article was first published in the October 2011 issue of the EURORDIS newsletter
Author: Paloma Tejada
Photo credits: © EURORDIS
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