The Voice of Rare Disease
Patients in Europe
Multi-stakeholder collaboration is crucial to improving access
“I dream that all patients throughout Europe and beyond, no matter the socio-economic background and no matter the complexity of the situation, will be able to access medicines and that these medicines are reimbursed.”
“I dream that this dream can come true one day. 15 years ago my dream was to have two new lungs to save my life. This dream came true. Dreams can come true.”
Pisana Ferrari, a patient advocate for over 15 years & Chief Executive Officer of PHA Europe, kicked off the 2nd Multi-Stakeholder Symposium on Improving Patients’ Access for Rare Diseases with this passionate plea.
Even though ever-advancing science and technology now create rapidly growing opportunities to develop more innovative medicines, the rare disease community is still facing problems accessing these medicines in Europe.
At the Symposium, EURORDIS brought together all stakeholders that play a role in getting medicines to rare disease patients to initiate a cooperative process that respects all of their interests and that will lead to solutions for improving patients’ access to medicines.
The ultimate goal of this process is to reach long‐lasting solutions that are affordable and sustainable for national healthcare systems, which in turn help improve patients’ access.
Participants included patient advocates, academics, clinicians, policymakers, investors, members of the EURORDIS Round Table of Companies and other representatives from the healthcare industry, as well as from payer and health-technology-assessment authorities.
Watch day 1 of the symposium, see the pre-reading documents and see the conversation on social media during the event via #RareEU2017.
The Symposium, which builds on the first Multi-Stakeholder Symposium on Improving Patients’ Access to Rare Disease Therapies in February 2016, created an open forum for all groups to express their concerns and interests in improving patients’ access to rare disease medicines.
Participants discussed existing initiatives aimed at improving access, such as the Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA).
Sessions focused on topics including the new European Reference Networks and their role in generating quality data, the future of EU-level cooperation on health technology assessment, as well as between payers and companies, pan-European disease registries, and innovative performance-based outcome agreements.
Participants also discussed nine new recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases to help improve the consistency of pricing and reimbursement for orphan medicinal products in Europe.
Action, not just words
EURORDIS Chief Executive Officer Yann Le Cam kicked the conference off by calling for a three to fivefold increase in the number of rare disease medicines approved by the European Medicines Agency and the US FDA per year by 2025 and for those medicines to be available at one third to one fifth of the price.
Patient representatives Elizabeth Vroom, President of the Dutch Duchenne Parent Project (who won the EURORDIS Volunteer Award the night before!) and Angela Paton, MPS Society UK, told their personal stories of involvement in medicines development, pricing and reimbursement processes.
Keynote speaker Xavier Prats Monné, Director General, Directorate General for Health and Food Safety at the European Commission, thanked EURORDIS and patient organisations for their work for the rare disease community.
He also commented on the new European Reference Networks, “We are confident that the ERN network will be a real demonstration of an area where the EU can do more.”
He concluded his keynote by saying, “I would like to stress three areas where we agree more needs to be done: one, make more effort towards a common approach to the value and the price of orphan medicines; two, make more effort towards a sustainable approach to evidence generation building on the ERNs; and three, we need a more trusted and transparent cooperation between payers and companies”.
He concluded, “Rare diseases are not about health only, they are about equity, they are about social justice, they are about inequalities.”
In the closing remarks for the Symposium, Yann Le Cam announced the imminent formation of a new multi-stakeholder group that will draft a OneText Collaborative Plan of Action for all parties to collaborate in a process to improve patients’ access to medicines. This process will aim to enhance and sustain trust between the various stakeholder groups, a fundamental condition to achieve patient access to medicines.
He congratulated participants on their dialogue and for identifying key issues in making medicines accessible, but called upon participants to be even more open in the future about their own interests, to propose more out-of-the-box options, and contribute to the identification of acceptable solutions enabling us to achieve the end goal, even if that presupposes changes they didn’t anticipate. We expect nothing less than radical change to improve the system.”
New EURORDIS reflection paper on access
EURORDIS and its members also launched a reflection paper “Breaking the Access Deadlock to Leave No One Behind – a contribution on possibilities for patients’ full and equitable access to rare disease therapies” at the Symposium.
This paper, still a work in progress, is EURORDIS’ first on the topic of access since the joint EURORDIS-EPF ‘Call on Payers’ in 2015. The paper sets out how to design a new and improved approach to ensure patients’ full access to these medicines. This approach should address the current imbalance and restore in parallel both transparency and trust between payers and pharmaceutical companies.
Eva Bearryman, Communications Manager, EURORDIS