Euronews has gathered a panel of experts, industry leaders and key players in Brussels to address the issue of how rare disease provision can be advanced in the EU.
In the European Union, a disease is considered rare if it affects fewer than five people in 10,000.
This might sound small but in practice, six to eight per cent of the bloc’s population is affected by a distinct rare condition like this, which translates to between 27 and 36 million people.
Most of these patients suffer from even rarer diseases that affect just one person in 100,000 or more, according to EU data.
Approximately 5,000-8,000 distinct rare diseases in the EU span all medical areas and include neurological, immunological, metabolic diseases, as well as rare cancers.
You can watch the debate live in this article on February 15 at 15:00 CET.
Guests joining the discussion will include Alessandra Moretti, Member of the European Parliament, Yann Le Cam, CEO of EURORDIS, and Johanna Friedl-Naderer, president of Europe, Canada and partner markets at Biogen. More information on attendees, including a fourth profile, will be disclosed in the run-up to the debate.
Debate host and Euronews science journalist Jeremy Wilks will accompany the panel as they consider rare diseases and the battle for progress, tackling questions like how the private and public sector can work better together and what the future formula for better healthcare should look like.
Over the last 20 years, innovation surrounding rare diseases in Europe has improved dramatically, with a specific framework put in place by the EU called the Orphan Medicinal Products Regulation.
This rule concerns products developed to treat medical conditions which, because they are so rare, would not be profitable to produce without supporting governmental measures.
Previously only eight orphan medicines were available for patients but this number has risen to more than 200, resulting in the pharmaceutical sector delivering treatments for up to 6.3 million patients.
Our panel will look at the challenge facing policymakers in devising a framework that improves the development of orphan medicinal products and how innovation in the medicines and treatments that are essential for those with rare diseases is notoriously challenging due to lack of knowledge and small patient population.
The panel will also consider the work and recommendations of the European Expert Group on Orphan Drug Incentives, an expert unit that brings together representatives of the broad rare disease community.
After coming together in 2020, the group’s goal is to develop policy proposals to facilitate EU policymakers and its members include researchers, academics, patient representatives, members of the investor community, individuals from rare disease companies and also trade associations.