Rare Disease Day, 29 February, marks a moment when an estimated 30 million people living with rare diseases across Europe gain a louder voice. Europe now has a generational opportunity to scale up programmes capable of improving millions of lives.
A disease is considered rare when it affects fewer than one in 2,000 people. 75% of rare diseases affect children, while 70% have their onset during childhood.
At the same time, over 90% of the more than 7,000 rare diseases do not have an approved treatment option, while 70% of people with rare diseases wait more than one year to get a confirmed diagnosis.
A new data-led, 360-comprehensive strategy is called for by rare disease experts. In this Special Report, we look at the issues affecting rare disease patients, and the emerging roadmap to improve the clinical pathway.
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Europe’s rare diseases postcode lottery creating health inequality
Est. 9minPatients suffering from rare diseases across the EU, and their families, are struggling on the road to lifesaving and life-altering healthcare. The difficult patient Odyssey is warped by a postcode lottery.
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Orphan medicines Pharma package provisions unleash uncertainties, stakeholders warn
Est. 8minThere are more than 6,000 rare diseases, with 27 different healthcare systems governed by diverse rules and principles, with differences for orphan drugs payments. The new Pharma Package aims on bridging regulatory gaps, but stakeholders warn of the risk of amplifying uncertainties.
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New rare diseases action plan on horizon, Europe at turning point
Est. 7minRare Disease Day, 29 February, marks a moment in which Europe is facing a generational opportunity to scale up programmes capable of improving millions of lives. A new data-led, 360-comprehensive strategy is called for by rare disease experts.
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Genetic diagnostic technology a game changer for rare diseases, but ethics concerns linger
Est. 8minGenetic diagnostic technology advances for rare diseases offer hope, but gene-tech comes wrapped in complex ethical concerns. Will Europe accelerate modern diagnostics, or will ethical concerns slow and limit innovation?
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Right here, right now European public health policies must start giving people living with rare diseases a fair chance
Est. 7minThis year’s Rare Disease Day (29 February) comes at a critical moment for European rare disease policy. Now more than ever, it’s essential that EU policies enable health equity for people living with rare diseases, says Alexion’s Soraya Bekkali.