ALPE at BioMarin European Patient Advocacy Forum 2018

On the 17th November 2018, Carmen Alonso represented ALPE

in a meeting organized by BioMarin aiming the discussion on "The Power of Effective Data-Driven Storytelling". The meeting took place in Amsterdam and had the participation of 28 patient representatives from several diseases groups in Europe and several BioMarin collaborators, mainly from the USA.

The overarching goal of this Forum was to increase patient representatives knowledge on patients data, that is usually collected by patient organizations and can be a crucial resource in advocating for social rights, legal aspects, medicines appraisal, and knowing better the patient community in a specific country and also at a more broader level, in case of federations.

Image 1.Topics covered during the Forum. Credits- Jan Geissler, Patvocates 


The moderators of the full event were Jan Geissler and Támas Bereczky, both from EUPATI and Patvocates. who did an excellent work to compel participants in joining all the moments of the share of ideas and debate.

 Image 2. Suggestions by participants on the key topics they wanted to see discussed


The colleagues from Turkey revealed serious social issues in having a voice in their country, in advocating for patients. It was clear that all organizations present had a very distinctive approach to advocacy, but it also depended on the political and social barriers already existent. So, many aspects are very difficult to deal with in rare diseases, but in many countries, the national system puts over that, many barriers for patient advocates.

How to Collect Data that Matter

Declan Noone, European Haemophilia Consortium and Project Coordinator of PARTNERS Programme give a very enthusiastic and a session on this title just presented, that is a very important theme for patient organizations to understand better their patient community and engage better with other relevant stakeholders in their country and at an international level.

So, Why do patient organizations need to collect data?

To make the advocacy case for improved treatment and care, to demonstrate the success of care, to defend the level of and resources for care in a difficult economic environment and run comparative data from other countries to assist in national advocacy.

As also something very relevant was the suggestions to clarify the patient groups advocacy work questioning what is the problem to be addressed? And what do you want to do about it? and having data is not really key if we don´t know exactly what we want them to do with it?

Jan Geissler also presented a real example of the benefits on data collection and what can be done with that valorous information with the session "Evidence-Based Advocacy: Introduction to Community-Led Data Collection", from the CML community experience. This was a very enriching session that put us considering all that we can do for patients using the data that we are given by them. Most important is that the data collected by patient groups must obey the GDPR data protection laws, so all data used must always be anonymized, that is saying with no identification of the patients.

Jan also presented how and when patients representatives can take action in medicines R&D, that is critical for rare diseases, once the knowledge about then in limited. Patients can add critical information in all these moments. 

Image 3. Patient expert participation in the medicines research & development roadmap. Credits: Jan Geissler, Patvocates

During the afternoon, was time for two Breakout Sessions on Creating an Action Plan for Data-Driven Storytelling. Both were very participated and the conclusions were strong indicators that patient organizations have a key role in advocating for patients and for using the patients' data for social, research and legal actions.

To finish, Hank Fuchs, MD, President, Worldwide Research & Development, presented the newest project of BioMarin "The Tree of life" towards putting patients in the heart of the company.

Images 4 & 5- The sculpture "Tree of life", by Gordon A Huether, that is now in BioMarin headquartered in San Rafael, California. Credits: CODAworx


Overall, this was a very positive meeting, where we all could hear colleagues from other countries and another group of diseases and learn with them together.

Our sincere Thank you to BioMarin for this effort in setting this empowering Forum for Patient Organizations.