Driving EU policy change for people living with rare diseases: a focus on Wilson’s disease

On February 28, 2023, Rare Disease Day, the European Liver Patients’ Association and the Wilson’s Disease Policy Network brought together key experts and stakeholders in Wilson’s disease, rare diseases and health policy.

A range of speakers drew on the experiences of people living with Wilson’s disease to discuss how gaps in care and policy barriers can impact health outcomes and quality of life. They also discussed key activities and initiatives at the EU level and explained the priority policy actions that are urgently needed to improve the lives of people living with rare conditions.

The event also marked the launch of the Policy brief on Wilson’s disease that was developed by the members of the multidisciplinary Wilson’s Disease Policy Network.

The event was hosted in Brussels by MEP Ljudmila Novak, who welcomed all the participants and gave an introductory overview of the importance of raising awareness about rare diseases and, in particular, about a rare liver disease such as Wilson’s Disease.

Sindee Weinbaum, the Leader of the European Liver Patients’ Association working group on rare liver diseases, took the floor and described the aim of the meeting and the role of Wilson’s disease Policy Network. Two patients’ testimonies followed the introduction. The first was by David Martín Miguel from the Asociación Española de Familiares y Enfermos de Wilson (Spanish Association for Wilson’s Disease Patients and Their Relatives). The second was by Caroline Roatta, from the Association Bernard Pépin in France. Both stressed the patient perspective and the role of patients in improving Wilson’s disease health outcome.

Karl Heinz Weiss, Associate Professor of Gastroenterology from the Salem Medical Center in Germany, analysed the barriers to best-practice care for people living with Wilson’s disease.

An insight into EU action on rare diseases with a focus on rare disease research was given by Hélène Le Borgne, representing the European Commission, Directorate-General for Research and Innovation.

The event was wrapped up by a panel discussion moderated by Jody Tate from the Wilson’s Disease Policy Network. Marko Korenjak, President of the European Liver Patients’ Association, Alexander Natz, Secretary-General of the European Confederation of Pharmaceutical Entrepreneurs and Alina Sennrepresenting the European Commission, Directorate-General for Health and Food Safety, joined Karl Heinz Weiss and David Martín Miguel to discuss priorities and actions to drive changes in rare disease policy at the EU level.

MEP Ljudmila Novak addressed her closing remarks to all the participants stressing the importance of collaboration among stakeholders to close the gaps in care and overcome policy barriers for Wilson’s disease patients.