Today, February 28, marks Rare Disease Day: an annual event to help highlight the effects of rare diseases on individuals, families and the community, and to raise awareness in the hope of finding treatments, or even cures. As a snapshot, a rare disease is defined in Europe as one which affects less than 1 in 200,000 people. Some 6,000-8,000 rare diseases have been identified to date, and it’s estimated that up to 30 million people across the European Union and another 30 million across the US are affected by a rare disease.
Clinuvel takes an active stance on patients’ rights – we’re working hard to get SCENESSE® approved for the rare light intolerance erythropoietic protoporphyria (EPP). This year’s theme Rare Diseases Without Borders resonates with us particularly, as it recognises the global challenges that rare disease patients, and those working with them, face. Cultural and linguistic challenges aside, to get a new drug to rare disease patients one must find those patients then tackle myriad national, regional and local laws and regulations every step of the way. In short, a more cohesive, multinational approach is needed to better address the needs of rare disease patients. (more…)