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A Watershed Moment for Rare Disease in Europe

It's the right time to work for consensus to improve the care of rare disease patients, CSL Behring leaders say.

A panel discussion from the European Health Summit

A golden opportunity exists in Europe to improve policies that impact the care of people who have rare diseases, CSL Behring leaders said following this summer’s European Health Summit.

“Now is the moment for strong EU response and strategic vision to support patients, systems and scientific progress,” said an article in Euractiv, a media outlet that focuses on EU policy matters.

Attendees were encouraged by discussions at the summit, particularly because EU Council Presidencies (France, Czechia, Sweden) put out an agenda that laid out clear intention to advance the response to rare diseases in Europe.

The article quoted Cédric Hermans, head of hematology at the Belgian Saint-Luc University Hospital, who said rapid scientific advances have amounted to a revolution in patient care.

“Treatments for rare diseases are not a cost, but an investment,” he said.

The article also quoted Antti Kourula, Global Vice-President for Commercial Operations at CSL Behring. She said “improving dialogue and interactions between regulators, national Health Technology Assessment (HTA) bodies and industry can support market entry of innovative therapies for the benefit of patients.”

Rare diseases represent a good case study of what can be done to improve healthcare systems, Kourula added. She was optimistic and offered this vision for rapid progress by the coming decade.

“Towards the horizon 2030, new ways of assessing innovative therapies will have been co-defined and we will have embraced the value of curing rare disease patients, versus chronic care. Patients will always be at the center of efforts to improve care pathways,” Kourula said.

Read the full article in Euractiv, which was jointly authored by Irene de Cara Torres, CSL Behring’s Head of Europe Policy, Advocacy, and Government Affairs and Arnaud Thysen, Director-General of the European Business Summit: Tackling rare disease in Europe towards the horizon 2030