Podcast: A Stronger Europe for Rare Diseases

The fifth episode of the “European Health Union – Now!” podcast series looks at the situation of rare disease patients in Europe, what it means to live on the wrong side of the border, why orphan drugs are a different kind of animal, and what collaborative partnerships are needed to improve the situation of patients.

Just a few facts and figures to paint a picture of the current situation:

· There are around 30 million rare disease patients in Europe.

· 50% of them are children

· 30% don’t live till their 5th birthday

· 95% of rare disease patients are without a treatment option altogether.

The development of orphan drugs is a financially risky and not very lucrative business for pharma companies. While a lot needs to be invested into research and development, 48% of orphan medicinal products (OMPs) in the EU have a revenue of less than 10 million Euros. On the other hand, also payers face difficult decisions on reimbursing often very expensive orphan therapies for a small patient population. Though the situation for patients has clearly improved since new European legislation was introduced in the year 2000 (the number of authorised OMPs has risen from 8 to 182), I think there is a consensus in the community that new models are needed for the treatment of rare disease patients, and the current OMP regulation review represents an opportunity to bridge at least some of these gaps.

In this episode Dorli Kahr-Gottlieb, Secretary General of the European Health Forum Gastein and co-initiator of the European Health Union initiative, speaks about this and much more with our guest , Dimitrios Athanasiou. Dimitrios has more than 25 years’ experience with international business projects, and has worked in various countries in consulting, developing and reorganising companies. When his son was diagnosed with Duchenne Muscular Dystrophy, an incurable rare disease, he became a passionate international patient expert and advocate for Duchenne and Rare Diseases. Dimitrios is a board member of the World Duchenne Organization (WDO) and the European Patients’ Forum, and he represents EURORDIS as a Pediatric Committee member in the European Medicines Agency. In these roles, Dimitrios promotes the rights of patients with rare diseases to have access to the best care possible and to new, safe and affordable drugs.

Listen to the episode here.