March 23, 2017

IPOPI attends Rare Disease Day Celebrations in Brussels

As in the past, IPOPI was pleased to actively take part in the Rare Diseases events organised in Brussels which included the Eurordis Black Pearl Gala Dinner and the 2nd Multi-Stakeholder Symposium on Improving Patients’ Access for Rare Diseases.

The Gala dinner featured the EURORDIS Awards which recognise the outstanding commitment and achievements of patient organisations, volunteers, companies, scientists, media and policymakers who have contributed to reducing the impact of rare diseases on people’s lives.
The 2017 winners were presented with their awards by the European Commissioner for Health and Food Safety Vytenis Andriukaitis and include:

  • Policy Maker Award – Frédérique Ries, Member of the European Parliament, Belgium
  • Volunteer Award – Elizabeth Vroom, Duchenne Parent Project, the Netherlands
  • Media Award – Aldo Soligno, Photographer, Rare Lives Project, Italy
  • Company Award – GSK (GlaxoSmithKline) – Rare Diseases
  • Scientific Award – Dr Lucia Monaco, Chief Scientific Officer, Fondazione Telethon, Italy
  • Patient Organisation Award – The Dravet Syndrome European Federation, Spain
  • Lifetime Achievement Award – Anders Olauson, Founder of the Ågrenska Centre, Sweden

On February 22-23 Johan Prévot, IPOPI’s Executive Director, participated in the 2nd Multi-Stakeholder Symposium on Improving Patients’ Access for Rare Diseases. The Symposium brought together numerous participants from various stakeholder groups to express their concerns and interests in improving patients’ access to rare disease medicines.

Key topics discussed during the meeting included the Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA), 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.

The Symposium called 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.

The meeting concluded with the announcement of a new multi-stakeholder group that will draft a Collaborative Action Plan for all parties to collaborate in a process to improve patients’ access to RD medicines.