The May 10-12 meeting is sponsored by Eurodis, the Paris-based group that defines itself as a “patient-driven alliance” of rare disease organizations, and will focus on “collaborative strategies” that best hold promise to “leave no-one behind.”
BioNews Services — publisher of this website — cover the conference from Vienna.
Panelists discussed a range of topics based on six themes: breakthrough medicines on the horizon, quality of life, the digital patient, structuring the research and diagnosis landscape, economic perspectives in rare diseases, and global rare equity.
The meeting also features speeches and keynote addresses by Terkel Andersen, the group’s president; Beate Hartinger-Klein, Austria’s federal minister of health and social affairs; and Martin Seychell, deputy director-general for health and food safety of the European Commission, among others.
Jill Bonjean, the group’s corporate relations director, spoke to BioNews during last week’s World Orphan Drug Congress USA in Oxon Hill, Maryland.
“Access, of course, is a very key issue. Our goal is to have three to five times more new rare disease therapies approved per year, three to five times cheaper than today, by 2025,” Bonjean said. “We know that’s imprecise, but this is what we’re looking for.
“We want to make sure that, with the potential of all this tremendous science that is coming, patients will actually get access to these drugs,” she added.
According to the Eurordis paper, access to expensive medicines for rare diseases has become more difficult for reasons that include:
Economic stagnation since Europe’s 2008 financial crisis, and its consequence on the labor market and fiscal revenues, which means less money available for social programs and institutions
Europe’s aging population, which increases the demand for care
The organization of healthcare systems, which must be revisited and adapted to today’s challenges
The rising cost of new therapies, all the way from the lab to marketing approval, and
The advent of innovative treatments for widespread conditions, marketed by manufacturers at prices higher than ever before
Bonjean said half of those attended the conference — which took place at the Messe Wien Congress Center — are patients and patient advocates. Another 25 percent are pharmaceutical industry executives, and the remaining 25 percent are researchers, politicians, journalists, and others.
“When Eurordis came into being 20 years ago, people had already heard of cystic fibrosis or hemophilia, but most rare diseases were completely invisible,” Bonjean said. “Creating this identity of a rare disease community allowed more diseases to be better known, like Angelman or Rett syndrome.”
Eurordis operates on an annual budget of about €5 million (about $6 million) and employs 40 people in Paris, as well as a few staffers at EU headquarters in Brussels and at a smaller office in Barcelona, Spain. About a quarter of that budget comes from EU grants, another quarter from the pharmaceutical industry, and most of the rest from member organizations.
That meeting, which is open to both members and non-members, focuses on RDI’s efforts to put rare diseases on the United Nations’ 2030 Sustainable Agenda.
