NEWS

Eurordis’ First Summer School for Patient Advocates

Are you thinking about participating in a clinical trial but would like to better understand how they work? Would you like to know how an orphan drug is developed - from pre-clinical studies to its post-marketing life? What are the steps involved in the drug regulatory process in Europe? What role you can play in these processes as a patient advocate? The answers to these questions and more were discussed at the first Eurordis Summer School for Patient Advocates. Over 4 days, participants interacted with officials from the European Medicines Agency (EMEA), researchers, and patient representatives, in order to learn from each other’s experiences regarding clinical trials and drug development.

The Summer School consisted of a mix of tutorials and formal presentations, with an interactive approach. Participants were divided into 4 groups, assigned one orphan drug each and sent a pedagogic pack prior to their arrival in Barcelona. The pack provided background information about the drug, the disease it was meant to treat, and the associated regulatory papers. This method allowed participants to link the theoretical discussions to a practical case study.

Dr. Markku Toivonen, (NDA, Finland) began the formal presentations with a session on evidence-based medicine through clinical research. He went on to explain the life cycle of a drug and described the processes involved in clinical trials. The remainder of the day was devoted to understanding statistics involved in clinical research. Throughout all sessions participants were provided with a glossary to assist with learning and understanding the main concepts and vocabulary used in clinical trials. The next morning, they were able to discuss the ethical aspects of clinical trials and get to know the standards used for informed consent for participation in Europe and the US with ds Eric Koster MA (METC MST, The Netherlands) and Mr. Rob Camp (ATAC, Spain).

In the afternoon, the sessions were about exceptions to the “Gold standard” of randomised, controlled trials, especially when dealing with orphan medicinal products and paediatric drugs. Since these products are naturally designed for small populations they are not tied to the same standards as common drugs. Discussions about the specificity of orphan drugs in the regulatory process continued with a session by Dr. Patrick Salmon (IMB, Ireland) where he explained in detail the different steps of the regulatory procedures in Europe, including compassionate use and accelerated review.

Throughout the Summer School, participants were able to discuss in small group sessions and then regroup to hear formal presentations and ask questions. “Each group was given several documents including press releases that they could have come across in their everyday life. They had to discuss these and relate them to the topic at hand,” explained Dr. Maria Mavris, Drug Development Programme Manager at Eurordis. “The problem-based learning technique used in the small group sessions was very successful. Most participants said they found the set up less intimidating and it gave them the opportunity to interact more freely.”

The second part of the programme was especially interesting for those patient advocates who are interested in learning more about the EU decision making process at the EMEA, and the involvement of patient representatives in the various scientific committees and working parties. Dr. Juan Garcia Burgos of the EMEA provided a general overview of EMEA activities and patient representatives’ roles in the different committees. The work performed by the COMP, SAWP, PDCO, CHMP, PCWP and the upcoming Committee for Advanced Therapies was presented by officials, professors and patient representatives currently serving in these EMEA committees

The last session presented the different Eurordis task forces that patient advocates can join if they want to contribute to strengthening patients’ positions in the fields of orphan drugs, paediatric drugs, transparency and access to drugs.

To date, Eurordis’ patient representatives interested in drug development had not had the opportunity to meet and interact. The Eurordis Summer School offered this unique opportunity with the aim that participants pass on this knowledge to other patient groups in their country. “I will try to "educate" more people in our association and cooperate with other associations, public health authorities and doctors in Greece to further improve the lives of people with rare disorders,” said Stamatina Maniolas from the Greek Association of People with Genetic Abnormalities (TOMELLON). "The Summer School proved to be an important source of information and an inspiring environment where people from different countries and different diseases felt like a family."

After 4 days of intense work, the Summer School concluded in the historical administrative building of the Hospital overlooking the Sagrada Familia and with a short tour of the site of the Hospital Santa Creu i Sant Pau that is over 600 years old.

The Eurordis Summer School for Patient Advocates was made possible thanks to the sponsorship of the Drug Information Association (DIA) Philanthropy Programme, the partnership and generous support of the Fundacio Doctor Robert, the partnership of AFM and INSERM, and the financial support of the Spanish Medicine Agency.

Watch out for next year’s session of the Summer School!

For more information contact: Dr Maria Mavris at maria.mavriseurordis.org

Author: Paloma Tejada
Photo credits: all photos © Eurordis except Participants III © Matevosyan